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Christopher Palmer has been an associate editor at MDedge News since 2017. When he's not tidying grammar, he writes short pieces about breaking FDA announcements and approvals, as well as journal articles. He proudly holds a BA in English and philosophy. Follow him on Twitter @cmacmpalm.
FDA approves ustekinumab for ulcerative colitis
. This is the human interleukin-12 and IL-23 antagonist’s fourth indication since it was first approved by the FDA in 2009.
The approval is based on findings from the phase 3 UNIFI clinical trial, which achieved its primary endpoint of clinical remission over the course of both induction and maintenance. The 8-week induction study saw 19% of patients achieve clinical remission and 58% demonstrating clinical response; at the 1 year mark, after 44 weeks of maintenance therapy, 43% of patients were in clinical remission without steroids. The trial also assessed a novel histologic-endoscopic mucosal improvement endpoint that examined cellular improvement through both histologic examination and images observed during colonoscopy; 17% of treated patients achieved this endpoint at week 8, and 44% had by 1 year.
“The FDA approval of Stelara for [ulcerative colitis] represents an exciting milestone, offering patients a new option that has demonstrated improvement of the histology and endoscopic appearance of the intestinal lining, while also offering patients the potential for response and remission without the need for steroids,” said William J. Sandborn, MD, chief of the division of gastroenterology and professor of medicine at University of California, San Diego, and a study investigator.
Because of ustekinumab’s effects on the immune system, it may increase the risk of serious infection; as such, patients and health care professionals should discuss any signs of infection before initiation and continue discussions afterward. There are also concerns about risks of cancers, serious allergic reactions, and lung inflammation.
Full prescribing information is available on the Janssen website, as is the full press release regarding the approval
. This is the human interleukin-12 and IL-23 antagonist’s fourth indication since it was first approved by the FDA in 2009.
The approval is based on findings from the phase 3 UNIFI clinical trial, which achieved its primary endpoint of clinical remission over the course of both induction and maintenance. The 8-week induction study saw 19% of patients achieve clinical remission and 58% demonstrating clinical response; at the 1 year mark, after 44 weeks of maintenance therapy, 43% of patients were in clinical remission without steroids. The trial also assessed a novel histologic-endoscopic mucosal improvement endpoint that examined cellular improvement through both histologic examination and images observed during colonoscopy; 17% of treated patients achieved this endpoint at week 8, and 44% had by 1 year.
“The FDA approval of Stelara for [ulcerative colitis] represents an exciting milestone, offering patients a new option that has demonstrated improvement of the histology and endoscopic appearance of the intestinal lining, while also offering patients the potential for response and remission without the need for steroids,” said William J. Sandborn, MD, chief of the division of gastroenterology and professor of medicine at University of California, San Diego, and a study investigator.
Because of ustekinumab’s effects on the immune system, it may increase the risk of serious infection; as such, patients and health care professionals should discuss any signs of infection before initiation and continue discussions afterward. There are also concerns about risks of cancers, serious allergic reactions, and lung inflammation.
Full prescribing information is available on the Janssen website, as is the full press release regarding the approval
. This is the human interleukin-12 and IL-23 antagonist’s fourth indication since it was first approved by the FDA in 2009.
The approval is based on findings from the phase 3 UNIFI clinical trial, which achieved its primary endpoint of clinical remission over the course of both induction and maintenance. The 8-week induction study saw 19% of patients achieve clinical remission and 58% demonstrating clinical response; at the 1 year mark, after 44 weeks of maintenance therapy, 43% of patients were in clinical remission without steroids. The trial also assessed a novel histologic-endoscopic mucosal improvement endpoint that examined cellular improvement through both histologic examination and images observed during colonoscopy; 17% of treated patients achieved this endpoint at week 8, and 44% had by 1 year.
“The FDA approval of Stelara for [ulcerative colitis] represents an exciting milestone, offering patients a new option that has demonstrated improvement of the histology and endoscopic appearance of the intestinal lining, while also offering patients the potential for response and remission without the need for steroids,” said William J. Sandborn, MD, chief of the division of gastroenterology and professor of medicine at University of California, San Diego, and a study investigator.
Because of ustekinumab’s effects on the immune system, it may increase the risk of serious infection; as such, patients and health care professionals should discuss any signs of infection before initiation and continue discussions afterward. There are also concerns about risks of cancers, serious allergic reactions, and lung inflammation.
Full prescribing information is available on the Janssen website, as is the full press release regarding the approval
Duloxetine ‘sprinkle’ launches for patients with difficulty swallowing
Drizalma Sprinkle (duloxetine delayed-release capsule) has launched for the treatment of various neuropsychiatric and pain disorders in patients with difficulty swallowing, according to a release from Sun Pharma. It can be swallowed whole, sprinkled on applesauce, or administered via nasogastric tube.
Difficulty swallowing affects approximately 30%-35% of long-term care residents, but the main alternative – crushing tablets – introduces risks of its own to the administration process.
This sprinkle is indicated for the treatment of major depressive disorder in adults, generalized anxiety disorder in patients aged 7 years and older, diabetic peripheral neuropathic pain in adults, and chronic musculoskeletal pain in adults. It was approved by the Food and Drug Administration for these indications July 19, 2019.
It carries a boxed warning for suicidal thoughts and behaviors. The most common adverse reactions (5% or more of treated participants and twice the incidence with placebo) were nausea, dry mouth, somnolence, constipation, decreased appetite, and hyperhidrosis. The full prescribing information can be found on the FDA website.
cpalmer@mdedge.com
Drizalma Sprinkle (duloxetine delayed-release capsule) has launched for the treatment of various neuropsychiatric and pain disorders in patients with difficulty swallowing, according to a release from Sun Pharma. It can be swallowed whole, sprinkled on applesauce, or administered via nasogastric tube.
Difficulty swallowing affects approximately 30%-35% of long-term care residents, but the main alternative – crushing tablets – introduces risks of its own to the administration process.
This sprinkle is indicated for the treatment of major depressive disorder in adults, generalized anxiety disorder in patients aged 7 years and older, diabetic peripheral neuropathic pain in adults, and chronic musculoskeletal pain in adults. It was approved by the Food and Drug Administration for these indications July 19, 2019.
It carries a boxed warning for suicidal thoughts and behaviors. The most common adverse reactions (5% or more of treated participants and twice the incidence with placebo) were nausea, dry mouth, somnolence, constipation, decreased appetite, and hyperhidrosis. The full prescribing information can be found on the FDA website.
cpalmer@mdedge.com
Drizalma Sprinkle (duloxetine delayed-release capsule) has launched for the treatment of various neuropsychiatric and pain disorders in patients with difficulty swallowing, according to a release from Sun Pharma. It can be swallowed whole, sprinkled on applesauce, or administered via nasogastric tube.
Difficulty swallowing affects approximately 30%-35% of long-term care residents, but the main alternative – crushing tablets – introduces risks of its own to the administration process.
This sprinkle is indicated for the treatment of major depressive disorder in adults, generalized anxiety disorder in patients aged 7 years and older, diabetic peripheral neuropathic pain in adults, and chronic musculoskeletal pain in adults. It was approved by the Food and Drug Administration for these indications July 19, 2019.
It carries a boxed warning for suicidal thoughts and behaviors. The most common adverse reactions (5% or more of treated participants and twice the incidence with placebo) were nausea, dry mouth, somnolence, constipation, decreased appetite, and hyperhidrosis. The full prescribing information can be found on the FDA website.
cpalmer@mdedge.com
FDA approves transdermal asenapine system for schizophrenia
The Food and Drug Administration has approved a transdermal asenapine delivery system (Secuado) for treatment of schizophrenia in adults, according to a release from Noven Pharmaceuticals.
The patch formulation is designed to deliver sustained concentrations of asenapine over 24-hour periods, so the system is a once-daily treatment. The efficacy and safety profile for children younger than 18 years is unknown.
The approval is based on an international, phase 3, double-blind, placebo-controlled study that included 616 adults with schizophrenia. The transdermal system achieved the study’s primary endpoint of statistically significant improvement at week 6 in the Positive and Negative Syndrome Scale, compared with placebo.
The safety profile of the system was consistent with the known profile of sublingual asenapine, and the most commonly observed adverse reactions were extrapyramidal disorder, application site reaction, and weight gain. The full prescribing information includes a boxed warning explaining that antipsychotics, such as asenapine, are associated with increased risk of death among elderly patients with dementia-related psychosis, an indication not approved for this transdermal asenapine-delivery system. Other warnings described in the prescribing information include neuroleptic malignant syndrome, tardive dyskinesia, and metabolic changes.
clinical professor of psychiatry and behavioral sciences at New York Medical College, in the release.
Noven is a subsidiary of Hisamitsu Pharmaceutical.
The Food and Drug Administration has approved a transdermal asenapine delivery system (Secuado) for treatment of schizophrenia in adults, according to a release from Noven Pharmaceuticals.
The patch formulation is designed to deliver sustained concentrations of asenapine over 24-hour periods, so the system is a once-daily treatment. The efficacy and safety profile for children younger than 18 years is unknown.
The approval is based on an international, phase 3, double-blind, placebo-controlled study that included 616 adults with schizophrenia. The transdermal system achieved the study’s primary endpoint of statistically significant improvement at week 6 in the Positive and Negative Syndrome Scale, compared with placebo.
The safety profile of the system was consistent with the known profile of sublingual asenapine, and the most commonly observed adverse reactions were extrapyramidal disorder, application site reaction, and weight gain. The full prescribing information includes a boxed warning explaining that antipsychotics, such as asenapine, are associated with increased risk of death among elderly patients with dementia-related psychosis, an indication not approved for this transdermal asenapine-delivery system. Other warnings described in the prescribing information include neuroleptic malignant syndrome, tardive dyskinesia, and metabolic changes.
clinical professor of psychiatry and behavioral sciences at New York Medical College, in the release.
Noven is a subsidiary of Hisamitsu Pharmaceutical.
The Food and Drug Administration has approved a transdermal asenapine delivery system (Secuado) for treatment of schizophrenia in adults, according to a release from Noven Pharmaceuticals.
The patch formulation is designed to deliver sustained concentrations of asenapine over 24-hour periods, so the system is a once-daily treatment. The efficacy and safety profile for children younger than 18 years is unknown.
The approval is based on an international, phase 3, double-blind, placebo-controlled study that included 616 adults with schizophrenia. The transdermal system achieved the study’s primary endpoint of statistically significant improvement at week 6 in the Positive and Negative Syndrome Scale, compared with placebo.
The safety profile of the system was consistent with the known profile of sublingual asenapine, and the most commonly observed adverse reactions were extrapyramidal disorder, application site reaction, and weight gain. The full prescribing information includes a boxed warning explaining that antipsychotics, such as asenapine, are associated with increased risk of death among elderly patients with dementia-related psychosis, an indication not approved for this transdermal asenapine-delivery system. Other warnings described in the prescribing information include neuroleptic malignant syndrome, tardive dyskinesia, and metabolic changes.
clinical professor of psychiatry and behavioral sciences at New York Medical College, in the release.
Noven is a subsidiary of Hisamitsu Pharmaceutical.
Schizophrenia patients have ‘aberrant’ response to Epstein-Barr
Individuals with schizophrenia had elevated levels of one Epstein-Barr virus (EBV) antibody but atypically low levels of another, which investigators deemed an “aberrant immune response.”
Faith Dickerson, PhD, MPH, Lorraine Jones-Brando, PhD,and colleagues investigated the IgG antibodies and genetics of 432 individuals with schizophrenia and 311 without. The investigators used solid-phase immunoassays to measure antibodies, and they measured titers of antibodies not just for EBV but also for related viruses. The group with schizophrenia was slightly older, with a mean age of 38.2 years, compared with a mean age of 32.03 years in the group without schizophrenia. Also, 65.7% of the study participants in the schizophrenia group were male, compared with 39.2% of those in the group without schizophrenia. More than 60% of participants in the schizophrenia group were cigarette smokers, compared with 14.8% of the controls. The study was published in Schizophrenia Bulletin.
Compared with the controls, individuals with schizophrenia had elevated levels of EBV viral capsid antibody (EBV-VCA) with a mean effect size of 0.356 (P less than .002); however, the levels of EBV nuclear antigen-1 (EBNA-1) were not significantly different from those seen in individuals without schizophrenia, reported Dr. Dickerson of the Stanley Research Program at Sheppard Pratt, and Dr. Jones-Brando of Johns Hopkins University, both in Baltimore.
The investigators also examined adjusted odds ratios for individuals with schizophrenia having levels of antibodies higher than percentile cutoffs of the controls; for example, the aOR for those individuals having EBV VCA levels at the 90th percentile of controls was 2.03 (95% confidence interval, 1.23-3.37; P = .007). The aORs for EBNA-1 were not significant.
Those results suggest an aberrant immune response to EBV because, in most cases, EBV VCA and EBNA-1 are expressed at roughly equal levels, the investigators said.
“An increased understanding of the role of EBV infection might thus lead to novel methods for the prevention and treatment of schizophrenia.”
The study was funded by the Silvio O. Conte Center at Johns Hopkins University in Baltimore, and the Stanley Medical Research Institute, Chevy Chase, Md. Dr. Dickerson, Dr. Jones-Brando, and colleagues reported having no conflicts of interest.
SOURCE: Dickerson F et al. Schizophr Bull. 2019 Sep 11;45(5):1112-9.
Individuals with schizophrenia had elevated levels of one Epstein-Barr virus (EBV) antibody but atypically low levels of another, which investigators deemed an “aberrant immune response.”
Faith Dickerson, PhD, MPH, Lorraine Jones-Brando, PhD,and colleagues investigated the IgG antibodies and genetics of 432 individuals with schizophrenia and 311 without. The investigators used solid-phase immunoassays to measure antibodies, and they measured titers of antibodies not just for EBV but also for related viruses. The group with schizophrenia was slightly older, with a mean age of 38.2 years, compared with a mean age of 32.03 years in the group without schizophrenia. Also, 65.7% of the study participants in the schizophrenia group were male, compared with 39.2% of those in the group without schizophrenia. More than 60% of participants in the schizophrenia group were cigarette smokers, compared with 14.8% of the controls. The study was published in Schizophrenia Bulletin.
Compared with the controls, individuals with schizophrenia had elevated levels of EBV viral capsid antibody (EBV-VCA) with a mean effect size of 0.356 (P less than .002); however, the levels of EBV nuclear antigen-1 (EBNA-1) were not significantly different from those seen in individuals without schizophrenia, reported Dr. Dickerson of the Stanley Research Program at Sheppard Pratt, and Dr. Jones-Brando of Johns Hopkins University, both in Baltimore.
The investigators also examined adjusted odds ratios for individuals with schizophrenia having levels of antibodies higher than percentile cutoffs of the controls; for example, the aOR for those individuals having EBV VCA levels at the 90th percentile of controls was 2.03 (95% confidence interval, 1.23-3.37; P = .007). The aORs for EBNA-1 were not significant.
Those results suggest an aberrant immune response to EBV because, in most cases, EBV VCA and EBNA-1 are expressed at roughly equal levels, the investigators said.
“An increased understanding of the role of EBV infection might thus lead to novel methods for the prevention and treatment of schizophrenia.”
The study was funded by the Silvio O. Conte Center at Johns Hopkins University in Baltimore, and the Stanley Medical Research Institute, Chevy Chase, Md. Dr. Dickerson, Dr. Jones-Brando, and colleagues reported having no conflicts of interest.
SOURCE: Dickerson F et al. Schizophr Bull. 2019 Sep 11;45(5):1112-9.
Individuals with schizophrenia had elevated levels of one Epstein-Barr virus (EBV) antibody but atypically low levels of another, which investigators deemed an “aberrant immune response.”
Faith Dickerson, PhD, MPH, Lorraine Jones-Brando, PhD,and colleagues investigated the IgG antibodies and genetics of 432 individuals with schizophrenia and 311 without. The investigators used solid-phase immunoassays to measure antibodies, and they measured titers of antibodies not just for EBV but also for related viruses. The group with schizophrenia was slightly older, with a mean age of 38.2 years, compared with a mean age of 32.03 years in the group without schizophrenia. Also, 65.7% of the study participants in the schizophrenia group were male, compared with 39.2% of those in the group without schizophrenia. More than 60% of participants in the schizophrenia group were cigarette smokers, compared with 14.8% of the controls. The study was published in Schizophrenia Bulletin.
Compared with the controls, individuals with schizophrenia had elevated levels of EBV viral capsid antibody (EBV-VCA) with a mean effect size of 0.356 (P less than .002); however, the levels of EBV nuclear antigen-1 (EBNA-1) were not significantly different from those seen in individuals without schizophrenia, reported Dr. Dickerson of the Stanley Research Program at Sheppard Pratt, and Dr. Jones-Brando of Johns Hopkins University, both in Baltimore.
The investigators also examined adjusted odds ratios for individuals with schizophrenia having levels of antibodies higher than percentile cutoffs of the controls; for example, the aOR for those individuals having EBV VCA levels at the 90th percentile of controls was 2.03 (95% confidence interval, 1.23-3.37; P = .007). The aORs for EBNA-1 were not significant.
Those results suggest an aberrant immune response to EBV because, in most cases, EBV VCA and EBNA-1 are expressed at roughly equal levels, the investigators said.
“An increased understanding of the role of EBV infection might thus lead to novel methods for the prevention and treatment of schizophrenia.”
The study was funded by the Silvio O. Conte Center at Johns Hopkins University in Baltimore, and the Stanley Medical Research Institute, Chevy Chase, Md. Dr. Dickerson, Dr. Jones-Brando, and colleagues reported having no conflicts of interest.
SOURCE: Dickerson F et al. Schizophr Bull. 2019 Sep 11;45(5):1112-9.
FROM SCHIZOPHRENIA BULLETIN
Patients with nonaffective psychosis offer treatment target preferences
‘Worrying less,’ ‘feeling happier,’ and sleeping better were among top targets identified
Patients with nonaffective psychosis want the “causal mechanisms” for their psychotic experiences treated, according to a study conducted by researchers at the University of Oxford (England).
“The findings indicate a need to adopt into services for people with severe mental health difficulties interventions shown by research to treat anxious avoidance, worry, low self-esteem, insomnia, and other such transdiagnostic mechanisms and evaluate the outcomes,” wrote Daniel Freeman, PhD, DClinPsy, of the department of psychiatry at Oxford, and colleagues. The study was published in Schizophrenia Research.
In previous research, Dr. Freeman and colleagues identified factors that appear to maintain symptoms of nonaffective psychosis, such as paranoia (Behav Cogn Psychother. 2016 Sep;44[5]:539-52).Those factors include anxious avoidance, worry, low self-esteem, insomnia, and reasoning bias. In additional research, the researchers demonstrated that addressing those targets can lead to led to reductions in paranoia.
In the current study, 1,809 patients were recruited from 39 National Health Services mental health trusts across England. The researchers analyzed responses to eight clinical assessment tools and a patient-reported questionnaire about treatment preferences.
Dr. Freeman and colleagues found consistency in the relationships observed among the factors and nonaffective psychosis with the theoretical model they had developed, such as positive correlations between paranoia and anxious avoidance (correlation coefficient [r] = 0.37), worry (r = 0.40), and hallucinations (r = 0.54). They found negative correlations between paranoia and levels of positive self-beliefs (r = –0.17), rational reasoning (r = –0.14), and psychological wellbeing (r = –0.29). Those correlations were all statistically significant with P values less than .001.
Most of the patients (90.3%) wanted at least one of the eight problem areas assessed in the study treated, and on average, they wanted more than four (mean, 4.5) treated. The most frequently identified top treatment priorities identified by patients were worrying (50.8%), feeling happier (42.9%), and sleeping better (41.2%).
the researchers wrote.
Among the study limitations: The assessments selected were based on the theoretical model the researchers had developed and therefore may not have captured all difficulties experienced by the patients. Furthermore, although the sample was large, it’s unclear whether it was truly representative because they were drawn from certain mental health services that, for example, patients with milder symptoms might not seek out.
Dr. Freeman is a cofounder and chief clinical officer of Oxford VR. He has received funding from the National Institute of Health Research, Medical Research Council, and Wellcome Trust. No other authors reported conflicts of interest.
SOURCE: Freeman D et al. Schizophr Res. 2019. doi: 10.1016/j.schres.2019.07.016.
‘Worrying less,’ ‘feeling happier,’ and sleeping better were among top targets identified
‘Worrying less,’ ‘feeling happier,’ and sleeping better were among top targets identified
Patients with nonaffective psychosis want the “causal mechanisms” for their psychotic experiences treated, according to a study conducted by researchers at the University of Oxford (England).
“The findings indicate a need to adopt into services for people with severe mental health difficulties interventions shown by research to treat anxious avoidance, worry, low self-esteem, insomnia, and other such transdiagnostic mechanisms and evaluate the outcomes,” wrote Daniel Freeman, PhD, DClinPsy, of the department of psychiatry at Oxford, and colleagues. The study was published in Schizophrenia Research.
In previous research, Dr. Freeman and colleagues identified factors that appear to maintain symptoms of nonaffective psychosis, such as paranoia (Behav Cogn Psychother. 2016 Sep;44[5]:539-52).Those factors include anxious avoidance, worry, low self-esteem, insomnia, and reasoning bias. In additional research, the researchers demonstrated that addressing those targets can lead to led to reductions in paranoia.
In the current study, 1,809 patients were recruited from 39 National Health Services mental health trusts across England. The researchers analyzed responses to eight clinical assessment tools and a patient-reported questionnaire about treatment preferences.
Dr. Freeman and colleagues found consistency in the relationships observed among the factors and nonaffective psychosis with the theoretical model they had developed, such as positive correlations between paranoia and anxious avoidance (correlation coefficient [r] = 0.37), worry (r = 0.40), and hallucinations (r = 0.54). They found negative correlations between paranoia and levels of positive self-beliefs (r = –0.17), rational reasoning (r = –0.14), and psychological wellbeing (r = –0.29). Those correlations were all statistically significant with P values less than .001.
Most of the patients (90.3%) wanted at least one of the eight problem areas assessed in the study treated, and on average, they wanted more than four (mean, 4.5) treated. The most frequently identified top treatment priorities identified by patients were worrying (50.8%), feeling happier (42.9%), and sleeping better (41.2%).
the researchers wrote.
Among the study limitations: The assessments selected were based on the theoretical model the researchers had developed and therefore may not have captured all difficulties experienced by the patients. Furthermore, although the sample was large, it’s unclear whether it was truly representative because they were drawn from certain mental health services that, for example, patients with milder symptoms might not seek out.
Dr. Freeman is a cofounder and chief clinical officer of Oxford VR. He has received funding from the National Institute of Health Research, Medical Research Council, and Wellcome Trust. No other authors reported conflicts of interest.
SOURCE: Freeman D et al. Schizophr Res. 2019. doi: 10.1016/j.schres.2019.07.016.
Patients with nonaffective psychosis want the “causal mechanisms” for their psychotic experiences treated, according to a study conducted by researchers at the University of Oxford (England).
“The findings indicate a need to adopt into services for people with severe mental health difficulties interventions shown by research to treat anxious avoidance, worry, low self-esteem, insomnia, and other such transdiagnostic mechanisms and evaluate the outcomes,” wrote Daniel Freeman, PhD, DClinPsy, of the department of psychiatry at Oxford, and colleagues. The study was published in Schizophrenia Research.
In previous research, Dr. Freeman and colleagues identified factors that appear to maintain symptoms of nonaffective psychosis, such as paranoia (Behav Cogn Psychother. 2016 Sep;44[5]:539-52).Those factors include anxious avoidance, worry, low self-esteem, insomnia, and reasoning bias. In additional research, the researchers demonstrated that addressing those targets can lead to led to reductions in paranoia.
In the current study, 1,809 patients were recruited from 39 National Health Services mental health trusts across England. The researchers analyzed responses to eight clinical assessment tools and a patient-reported questionnaire about treatment preferences.
Dr. Freeman and colleagues found consistency in the relationships observed among the factors and nonaffective psychosis with the theoretical model they had developed, such as positive correlations between paranoia and anxious avoidance (correlation coefficient [r] = 0.37), worry (r = 0.40), and hallucinations (r = 0.54). They found negative correlations between paranoia and levels of positive self-beliefs (r = –0.17), rational reasoning (r = –0.14), and psychological wellbeing (r = –0.29). Those correlations were all statistically significant with P values less than .001.
Most of the patients (90.3%) wanted at least one of the eight problem areas assessed in the study treated, and on average, they wanted more than four (mean, 4.5) treated. The most frequently identified top treatment priorities identified by patients were worrying (50.8%), feeling happier (42.9%), and sleeping better (41.2%).
the researchers wrote.
Among the study limitations: The assessments selected were based on the theoretical model the researchers had developed and therefore may not have captured all difficulties experienced by the patients. Furthermore, although the sample was large, it’s unclear whether it was truly representative because they were drawn from certain mental health services that, for example, patients with milder symptoms might not seek out.
Dr. Freeman is a cofounder and chief clinical officer of Oxford VR. He has received funding from the National Institute of Health Research, Medical Research Council, and Wellcome Trust. No other authors reported conflicts of interest.
SOURCE: Freeman D et al. Schizophr Res. 2019. doi: 10.1016/j.schres.2019.07.016.
FROM SCHIZOPHRENIA RESEARCH
FDA approves afamelanotide for treatment of rare condition with light-induced pain
The Food and Drug Administration has approved , a rare condition that causes extremely painful reactions when skin is exposed to light, according to an FDA announcement.
This is the first treatment approved to help patients with this condition increase their exposure to light, according to the release.
Afamelanotide, administered in a subcutaneous implant, is a melanocortin-1 receptor (MC1-R) agonist, which “increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources,” the release says.
Approval is based on a pair of parallel-group clinical trials that compared the number of hours spent in sunlight in the treatment and placebo groups. The first trial enrolled 93 patients; 48 received afamelanotide. The treated patients spent a median of 61 hours in total over 180 days in direct sunlight between 10 a.m. and 6 p.m. on days with no pain, compared with 41 hours for patients taking placebo.
The second trial assessed the total number of hours over 270 days spent outdoors between 10 a.m. and 3 p.m. on days with no pain for which “most of the day” was spent in direct sunlight. In this study, 38 patients treated with afamelanotide spent a median total of 6 hours, compared with 0.75 hours among the remaining 36 who were taking a placebo.
The most common side effects include implant site reaction, nausea, and oropharyngeal pain. The implant should be administered only by trained professionals. Because afamelanotide may cause skin darkening, it’s recommended that patients should undergo twice-yearly skin examinations. Patients are also encouraged to maintain sun protection measures to help prevent phototoxic reactions.
“Today’s approval is one example of the FDA’s ongoing commitment to encourage industry innovation of therapies to treat rare diseases, and work with drug developers to make promising new therapies available to patients as safely and efficiently as possible,” said Julie Beitz, MD, director of FDA’s Center for Drug Evaluation and Research Office of Drug Evaluation III in the FDA release.
The Food and Drug Administration has approved , a rare condition that causes extremely painful reactions when skin is exposed to light, according to an FDA announcement.
This is the first treatment approved to help patients with this condition increase their exposure to light, according to the release.
Afamelanotide, administered in a subcutaneous implant, is a melanocortin-1 receptor (MC1-R) agonist, which “increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources,” the release says.
Approval is based on a pair of parallel-group clinical trials that compared the number of hours spent in sunlight in the treatment and placebo groups. The first trial enrolled 93 patients; 48 received afamelanotide. The treated patients spent a median of 61 hours in total over 180 days in direct sunlight between 10 a.m. and 6 p.m. on days with no pain, compared with 41 hours for patients taking placebo.
The second trial assessed the total number of hours over 270 days spent outdoors between 10 a.m. and 3 p.m. on days with no pain for which “most of the day” was spent in direct sunlight. In this study, 38 patients treated with afamelanotide spent a median total of 6 hours, compared with 0.75 hours among the remaining 36 who were taking a placebo.
The most common side effects include implant site reaction, nausea, and oropharyngeal pain. The implant should be administered only by trained professionals. Because afamelanotide may cause skin darkening, it’s recommended that patients should undergo twice-yearly skin examinations. Patients are also encouraged to maintain sun protection measures to help prevent phototoxic reactions.
“Today’s approval is one example of the FDA’s ongoing commitment to encourage industry innovation of therapies to treat rare diseases, and work with drug developers to make promising new therapies available to patients as safely and efficiently as possible,” said Julie Beitz, MD, director of FDA’s Center for Drug Evaluation and Research Office of Drug Evaluation III in the FDA release.
The Food and Drug Administration has approved , a rare condition that causes extremely painful reactions when skin is exposed to light, according to an FDA announcement.
This is the first treatment approved to help patients with this condition increase their exposure to light, according to the release.
Afamelanotide, administered in a subcutaneous implant, is a melanocortin-1 receptor (MC1-R) agonist, which “increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources,” the release says.
Approval is based on a pair of parallel-group clinical trials that compared the number of hours spent in sunlight in the treatment and placebo groups. The first trial enrolled 93 patients; 48 received afamelanotide. The treated patients spent a median of 61 hours in total over 180 days in direct sunlight between 10 a.m. and 6 p.m. on days with no pain, compared with 41 hours for patients taking placebo.
The second trial assessed the total number of hours over 270 days spent outdoors between 10 a.m. and 3 p.m. on days with no pain for which “most of the day” was spent in direct sunlight. In this study, 38 patients treated with afamelanotide spent a median total of 6 hours, compared with 0.75 hours among the remaining 36 who were taking a placebo.
The most common side effects include implant site reaction, nausea, and oropharyngeal pain. The implant should be administered only by trained professionals. Because afamelanotide may cause skin darkening, it’s recommended that patients should undergo twice-yearly skin examinations. Patients are also encouraged to maintain sun protection measures to help prevent phototoxic reactions.
“Today’s approval is one example of the FDA’s ongoing commitment to encourage industry innovation of therapies to treat rare diseases, and work with drug developers to make promising new therapies available to patients as safely and efficiently as possible,” said Julie Beitz, MD, director of FDA’s Center for Drug Evaluation and Research Office of Drug Evaluation III in the FDA release.
Interventions significantly improve NICU immunization rates
according to a study in Pediatrics.
Investigators led by Raymond C. Stetson, MD, of the Mayo Clinic in Rochester, Minn., identified three root causes of underimmunization in a NICU at Mayo Clinic: providers’ lack of knowledge about recommended immunization schedules; immunizations not being ordered when they were due; and parental hesitancy toward vaccination. They addressed these causes with the following five phases of intervention: an intranet resource educating providers about vaccine schedules and dosing intervals; a spreadsheet-based checklist to track and flag immunization status; an intranet resource aimed at discussion with vaccine-hesitant parents; education about safety in providing immunization and review of material from the first three interventions; and education about documentation, including parental consent.
Over the project period, 1,242 infants were discharged or transferred from the NICU. The study included a 6-month “improve phase,” during which interventions were implemented, and a “control phase,” during which the ongoing effects after implementation were observed. At baseline, the rate of fully immunized infants in the NICU was only 56% by time of discharge or transfer, but during the combined improve and control phases, it was 93% with a P value of less than .001.
One of the limitations of the study is that the first three interventions were introduced simultaneously, which makes it hard to determine how much effect each might have had.
“Infants treated in NICUs represent a vulnerable population with the potential for high morbidity and mortality from vaccine-preventable infections,” the investigators wrote. “Our [quality improvement] effort, and others, demonstrate that this population is at risk for underimmunization and that immunization rates can be improved with a small number of interventions. Additionally, we were able to significantly decrease the number of days that immunizations were delayed compared to the routine infant vaccination schedule.”
There was no external funding for the study. One of the coauthors is on safety committees of vaccine studies for Merck. The other authors have no relevant financial disclosures.
SOURCE: Stetson R et al. Pediatr. 2019. doi: 10.1542/peds.2019-0337.
according to a study in Pediatrics.
Investigators led by Raymond C. Stetson, MD, of the Mayo Clinic in Rochester, Minn., identified three root causes of underimmunization in a NICU at Mayo Clinic: providers’ lack of knowledge about recommended immunization schedules; immunizations not being ordered when they were due; and parental hesitancy toward vaccination. They addressed these causes with the following five phases of intervention: an intranet resource educating providers about vaccine schedules and dosing intervals; a spreadsheet-based checklist to track and flag immunization status; an intranet resource aimed at discussion with vaccine-hesitant parents; education about safety in providing immunization and review of material from the first three interventions; and education about documentation, including parental consent.
Over the project period, 1,242 infants were discharged or transferred from the NICU. The study included a 6-month “improve phase,” during which interventions were implemented, and a “control phase,” during which the ongoing effects after implementation were observed. At baseline, the rate of fully immunized infants in the NICU was only 56% by time of discharge or transfer, but during the combined improve and control phases, it was 93% with a P value of less than .001.
One of the limitations of the study is that the first three interventions were introduced simultaneously, which makes it hard to determine how much effect each might have had.
“Infants treated in NICUs represent a vulnerable population with the potential for high morbidity and mortality from vaccine-preventable infections,” the investigators wrote. “Our [quality improvement] effort, and others, demonstrate that this population is at risk for underimmunization and that immunization rates can be improved with a small number of interventions. Additionally, we were able to significantly decrease the number of days that immunizations were delayed compared to the routine infant vaccination schedule.”
There was no external funding for the study. One of the coauthors is on safety committees of vaccine studies for Merck. The other authors have no relevant financial disclosures.
SOURCE: Stetson R et al. Pediatr. 2019. doi: 10.1542/peds.2019-0337.
according to a study in Pediatrics.
Investigators led by Raymond C. Stetson, MD, of the Mayo Clinic in Rochester, Minn., identified three root causes of underimmunization in a NICU at Mayo Clinic: providers’ lack of knowledge about recommended immunization schedules; immunizations not being ordered when they were due; and parental hesitancy toward vaccination. They addressed these causes with the following five phases of intervention: an intranet resource educating providers about vaccine schedules and dosing intervals; a spreadsheet-based checklist to track and flag immunization status; an intranet resource aimed at discussion with vaccine-hesitant parents; education about safety in providing immunization and review of material from the first three interventions; and education about documentation, including parental consent.
Over the project period, 1,242 infants were discharged or transferred from the NICU. The study included a 6-month “improve phase,” during which interventions were implemented, and a “control phase,” during which the ongoing effects after implementation were observed. At baseline, the rate of fully immunized infants in the NICU was only 56% by time of discharge or transfer, but during the combined improve and control phases, it was 93% with a P value of less than .001.
One of the limitations of the study is that the first three interventions were introduced simultaneously, which makes it hard to determine how much effect each might have had.
“Infants treated in NICUs represent a vulnerable population with the potential for high morbidity and mortality from vaccine-preventable infections,” the investigators wrote. “Our [quality improvement] effort, and others, demonstrate that this population is at risk for underimmunization and that immunization rates can be improved with a small number of interventions. Additionally, we were able to significantly decrease the number of days that immunizations were delayed compared to the routine infant vaccination schedule.”
There was no external funding for the study. One of the coauthors is on safety committees of vaccine studies for Merck. The other authors have no relevant financial disclosures.
SOURCE: Stetson R et al. Pediatr. 2019. doi: 10.1542/peds.2019-0337.
FROM PEDIATRICS
Secukinumab continues promising axial spondyloarthritis results in PREVENT
New results for secukinumab (Cosentyx) in the PREVENT trial continue its promising trend for treatment of the full spectrum of axial spondyloarthritis (axSpA), according to a release from Novartis. The PREVENT trial is assessing this therapy in nonradiographic axSpA (nr-axSpA); it’s already approved in ankylosing spondylitis.
Results of 16-week data were presented in mid-September, and the new results represent 52-week data. Full details and data will be revealed at an upcoming scientific congress.
The PREVENT trial is an ongoing, 2-year trial with a 2-year extension phase; it enrolled 550 men and women with nr-axSpA and assessed their response to secukinumab with an induction phase, to it without induction, or to placebo. The trial met its primary endpoint of 40% improvement in Assessment in Spondyloarthritis International Society (ASAS40) criteria at 16 and 52 weeks, which indicates meaningful and sustained improvement. ASAS40 is defined as an improvement of at least 40% and both 10 out of 100 points in three of four domains (patient global assessment, pain, function, and inflammation) and no worsening in the remaining one. The safety profile in this trial was consistent with those seen in previous trials.
Overall, the most common adverse reactions seen with this interleukin-17A antagonist are nasopharyngitis, diarrhea, and upper respiratory tract infections. Patients should be evaluated for tuberculosis before initiating treatment. Serious infections have been observed, so be mindful of patients with chronic infections and discontinue secukinumab in patients who develop infections. Caution should be exercised when prescribing to patients with inflammatory bowel disease. Full prescribing information can be found on the Novartis website.
New results for secukinumab (Cosentyx) in the PREVENT trial continue its promising trend for treatment of the full spectrum of axial spondyloarthritis (axSpA), according to a release from Novartis. The PREVENT trial is assessing this therapy in nonradiographic axSpA (nr-axSpA); it’s already approved in ankylosing spondylitis.
Results of 16-week data were presented in mid-September, and the new results represent 52-week data. Full details and data will be revealed at an upcoming scientific congress.
The PREVENT trial is an ongoing, 2-year trial with a 2-year extension phase; it enrolled 550 men and women with nr-axSpA and assessed their response to secukinumab with an induction phase, to it without induction, or to placebo. The trial met its primary endpoint of 40% improvement in Assessment in Spondyloarthritis International Society (ASAS40) criteria at 16 and 52 weeks, which indicates meaningful and sustained improvement. ASAS40 is defined as an improvement of at least 40% and both 10 out of 100 points in three of four domains (patient global assessment, pain, function, and inflammation) and no worsening in the remaining one. The safety profile in this trial was consistent with those seen in previous trials.
Overall, the most common adverse reactions seen with this interleukin-17A antagonist are nasopharyngitis, diarrhea, and upper respiratory tract infections. Patients should be evaluated for tuberculosis before initiating treatment. Serious infections have been observed, so be mindful of patients with chronic infections and discontinue secukinumab in patients who develop infections. Caution should be exercised when prescribing to patients with inflammatory bowel disease. Full prescribing information can be found on the Novartis website.
New results for secukinumab (Cosentyx) in the PREVENT trial continue its promising trend for treatment of the full spectrum of axial spondyloarthritis (axSpA), according to a release from Novartis. The PREVENT trial is assessing this therapy in nonradiographic axSpA (nr-axSpA); it’s already approved in ankylosing spondylitis.
Results of 16-week data were presented in mid-September, and the new results represent 52-week data. Full details and data will be revealed at an upcoming scientific congress.
The PREVENT trial is an ongoing, 2-year trial with a 2-year extension phase; it enrolled 550 men and women with nr-axSpA and assessed their response to secukinumab with an induction phase, to it without induction, or to placebo. The trial met its primary endpoint of 40% improvement in Assessment in Spondyloarthritis International Society (ASAS40) criteria at 16 and 52 weeks, which indicates meaningful and sustained improvement. ASAS40 is defined as an improvement of at least 40% and both 10 out of 100 points in three of four domains (patient global assessment, pain, function, and inflammation) and no worsening in the remaining one. The safety profile in this trial was consistent with those seen in previous trials.
Overall, the most common adverse reactions seen with this interleukin-17A antagonist are nasopharyngitis, diarrhea, and upper respiratory tract infections. Patients should be evaluated for tuberculosis before initiating treatment. Serious infections have been observed, so be mindful of patients with chronic infections and discontinue secukinumab in patients who develop infections. Caution should be exercised when prescribing to patients with inflammatory bowel disease. Full prescribing information can be found on the Novartis website.
FDA approves benralizumab autoinjector for eosinophilic asthma
according to a press release from AstraZeneca. Benralizumab is already approved as add-on maintenance for this form of asthma, but not for other eosinophilic conditions or for acute bronchospasm or status asthmaticus.
The autoinjector “pen” was tested for usability and pharmacokinetic exposure in two studies, the phase 3 GRECO trial and the phase 1 AMES trial, respectively. The multicenter, open-label GRECO trial was designed to assess patient- or caregiver-reported functionality, and it found that 97% of at-home administrations were successful at week 12 and week 16. The multicenter, randomized, open-label, parallel-group AMES trial compared pharmacokinetic exposure with the subcutaneous administration using either prefilled syringe or prefilled autoinjector; it found that the eosinophils were rapidly depleted in patients with use of either device.
The safety profiles in both trials were comparable to those seen in previous trials. Hypersensitivity reactions have been sometimes observed in the hours following administration of benralizumab; discontinuation is advised in case of any hypersensitivity reaction. The therapy should not be used to treat acute asthma symptoms, such as exacerbations, or bronchospasm, and any reduction in corticosteroid therapy should be gradual and performed under careful supervision of a health care professional. Although benralizumab’s effects on helminth infections are currently unknown, care should be taken with preexisting or incident infections.
Full prescribing information can be found on the AstraZeneca website.
according to a press release from AstraZeneca. Benralizumab is already approved as add-on maintenance for this form of asthma, but not for other eosinophilic conditions or for acute bronchospasm or status asthmaticus.
The autoinjector “pen” was tested for usability and pharmacokinetic exposure in two studies, the phase 3 GRECO trial and the phase 1 AMES trial, respectively. The multicenter, open-label GRECO trial was designed to assess patient- or caregiver-reported functionality, and it found that 97% of at-home administrations were successful at week 12 and week 16. The multicenter, randomized, open-label, parallel-group AMES trial compared pharmacokinetic exposure with the subcutaneous administration using either prefilled syringe or prefilled autoinjector; it found that the eosinophils were rapidly depleted in patients with use of either device.
The safety profiles in both trials were comparable to those seen in previous trials. Hypersensitivity reactions have been sometimes observed in the hours following administration of benralizumab; discontinuation is advised in case of any hypersensitivity reaction. The therapy should not be used to treat acute asthma symptoms, such as exacerbations, or bronchospasm, and any reduction in corticosteroid therapy should be gradual and performed under careful supervision of a health care professional. Although benralizumab’s effects on helminth infections are currently unknown, care should be taken with preexisting or incident infections.
Full prescribing information can be found on the AstraZeneca website.
according to a press release from AstraZeneca. Benralizumab is already approved as add-on maintenance for this form of asthma, but not for other eosinophilic conditions or for acute bronchospasm or status asthmaticus.
The autoinjector “pen” was tested for usability and pharmacokinetic exposure in two studies, the phase 3 GRECO trial and the phase 1 AMES trial, respectively. The multicenter, open-label GRECO trial was designed to assess patient- or caregiver-reported functionality, and it found that 97% of at-home administrations were successful at week 12 and week 16. The multicenter, randomized, open-label, parallel-group AMES trial compared pharmacokinetic exposure with the subcutaneous administration using either prefilled syringe or prefilled autoinjector; it found that the eosinophils were rapidly depleted in patients with use of either device.
The safety profiles in both trials were comparable to those seen in previous trials. Hypersensitivity reactions have been sometimes observed in the hours following administration of benralizumab; discontinuation is advised in case of any hypersensitivity reaction. The therapy should not be used to treat acute asthma symptoms, such as exacerbations, or bronchospasm, and any reduction in corticosteroid therapy should be gradual and performed under careful supervision of a health care professional. Although benralizumab’s effects on helminth infections are currently unknown, care should be taken with preexisting or incident infections.
Full prescribing information can be found on the AstraZeneca website.
FDA expands Dysport’s upper-limb spasticity indication to children
The Food and Drug Administration has expanded the indication of abobotulinumtoxinA (Dysport) for upper-limb spasticity to include patients aged 2 years and older, according to a release from Ipsen. This botulinum toxin product received approval for this indication in adults in 2015 and approval for lower-limb spasticity in patients aged 2 years and older in 2016. Notably, Orphan Drug Exclusivity prevents it from being indicated for patients with cerebral palsy because another botulinum toxin product, onabotulinumtoxinA (Botox), already was approved for the indication in June 2019.
Spasticity affects the muscles and joints of extremities, especially in growing children, and is usually caused by nerve damage, such as head trauma or spinal cord injury. The degree of spasticity can vary from mild muscle stiffness to severe, painful, and uncontrollable muscle spasms.
AbobotulinumtoxinA was evaluated for upper-limb spasticity in a phase 3, randomized, double-blind, low-dose controlled, multicenter study; the study enrolled 210 children aged 2-17 years with the condition and a Modified Ashworth Scale grade 2 or greater for elbow and wrist flexors. The children were randomized 1:1:1 to injections of either 8 units/kg, 16 units/kg, or 2 units/kg into the elbow flexors and wrist flexors. At 6 weeks, there were statistically significant improvements in Modified Ashworth Scale grade, the primary endpoint, with least-square mean changes from baseline of –2.0, –2.3, and –1.6, respectively.
AbobotulinumtoxinA and all other botulinum toxin products carry a boxed warning, the most serious warning the FDA issues. This warning refers to risk of botulism-like symptoms caused by the botulinum toxin spreading away from the injection area; these symptoms can included sometimes life-threatening difficulty swallowing or breathing. AbobotulinumtoxinA is contraindicated in patients with known hypersensitivity to any botulinum toxin or any of the components, those with presence of infection at proposed injection site(s), and those with known allergy to cow’s milk protein. It is also important to note that botulinum toxin preparations are not interchangeable; the potency units of one are not the same as those of another. Full prescribing information can be found on the Ipsen website.
The Food and Drug Administration has expanded the indication of abobotulinumtoxinA (Dysport) for upper-limb spasticity to include patients aged 2 years and older, according to a release from Ipsen. This botulinum toxin product received approval for this indication in adults in 2015 and approval for lower-limb spasticity in patients aged 2 years and older in 2016. Notably, Orphan Drug Exclusivity prevents it from being indicated for patients with cerebral palsy because another botulinum toxin product, onabotulinumtoxinA (Botox), already was approved for the indication in June 2019.
Spasticity affects the muscles and joints of extremities, especially in growing children, and is usually caused by nerve damage, such as head trauma or spinal cord injury. The degree of spasticity can vary from mild muscle stiffness to severe, painful, and uncontrollable muscle spasms.
AbobotulinumtoxinA was evaluated for upper-limb spasticity in a phase 3, randomized, double-blind, low-dose controlled, multicenter study; the study enrolled 210 children aged 2-17 years with the condition and a Modified Ashworth Scale grade 2 or greater for elbow and wrist flexors. The children were randomized 1:1:1 to injections of either 8 units/kg, 16 units/kg, or 2 units/kg into the elbow flexors and wrist flexors. At 6 weeks, there were statistically significant improvements in Modified Ashworth Scale grade, the primary endpoint, with least-square mean changes from baseline of –2.0, –2.3, and –1.6, respectively.
AbobotulinumtoxinA and all other botulinum toxin products carry a boxed warning, the most serious warning the FDA issues. This warning refers to risk of botulism-like symptoms caused by the botulinum toxin spreading away from the injection area; these symptoms can included sometimes life-threatening difficulty swallowing or breathing. AbobotulinumtoxinA is contraindicated in patients with known hypersensitivity to any botulinum toxin or any of the components, those with presence of infection at proposed injection site(s), and those with known allergy to cow’s milk protein. It is also important to note that botulinum toxin preparations are not interchangeable; the potency units of one are not the same as those of another. Full prescribing information can be found on the Ipsen website.
The Food and Drug Administration has expanded the indication of abobotulinumtoxinA (Dysport) for upper-limb spasticity to include patients aged 2 years and older, according to a release from Ipsen. This botulinum toxin product received approval for this indication in adults in 2015 and approval for lower-limb spasticity in patients aged 2 years and older in 2016. Notably, Orphan Drug Exclusivity prevents it from being indicated for patients with cerebral palsy because another botulinum toxin product, onabotulinumtoxinA (Botox), already was approved for the indication in June 2019.
Spasticity affects the muscles and joints of extremities, especially in growing children, and is usually caused by nerve damage, such as head trauma or spinal cord injury. The degree of spasticity can vary from mild muscle stiffness to severe, painful, and uncontrollable muscle spasms.
AbobotulinumtoxinA was evaluated for upper-limb spasticity in a phase 3, randomized, double-blind, low-dose controlled, multicenter study; the study enrolled 210 children aged 2-17 years with the condition and a Modified Ashworth Scale grade 2 or greater for elbow and wrist flexors. The children were randomized 1:1:1 to injections of either 8 units/kg, 16 units/kg, or 2 units/kg into the elbow flexors and wrist flexors. At 6 weeks, there were statistically significant improvements in Modified Ashworth Scale grade, the primary endpoint, with least-square mean changes from baseline of –2.0, –2.3, and –1.6, respectively.
AbobotulinumtoxinA and all other botulinum toxin products carry a boxed warning, the most serious warning the FDA issues. This warning refers to risk of botulism-like symptoms caused by the botulinum toxin spreading away from the injection area; these symptoms can included sometimes life-threatening difficulty swallowing or breathing. AbobotulinumtoxinA is contraindicated in patients with known hypersensitivity to any botulinum toxin or any of the components, those with presence of infection at proposed injection site(s), and those with known allergy to cow’s milk protein. It is also important to note that botulinum toxin preparations are not interchangeable; the potency units of one are not the same as those of another. Full prescribing information can be found on the Ipsen website.