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Safety alert for intragastric balloon systems
The Food and Drug Administration announced a safety alert on Aug. 10, 2017, for liquid-filled intragastric balloon systems, as they have caused five reports of unanticipated deaths that occurred from 2016 to present in patients.
The cause or incidence of patient death is still unknown, and the FDA has not been able to definitively attribute the deaths to the devices or the insertion procedures for these devices. All five reports show that patient deaths occurred within a month or less of balloon placement. In three of the reports, death occurred as soon as 1-3 days after balloon placement. The FDA has also received two additional reports of deaths in the same time period related to potential complications associated with balloon treatment.
The FDA continues to recommend that health care providers closely monitor patients treated with these devices for complications. Any adverse events related to intragastric balloon systems should be reported through MedWatch. The FDA will keep the public informed as new information becomes available.
Read the full safety alert on the FDA’s website.
The Food and Drug Administration announced a safety alert on Aug. 10, 2017, for liquid-filled intragastric balloon systems, as they have caused five reports of unanticipated deaths that occurred from 2016 to present in patients.
The cause or incidence of patient death is still unknown, and the FDA has not been able to definitively attribute the deaths to the devices or the insertion procedures for these devices. All five reports show that patient deaths occurred within a month or less of balloon placement. In three of the reports, death occurred as soon as 1-3 days after balloon placement. The FDA has also received two additional reports of deaths in the same time period related to potential complications associated with balloon treatment.
The FDA continues to recommend that health care providers closely monitor patients treated with these devices for complications. Any adverse events related to intragastric balloon systems should be reported through MedWatch. The FDA will keep the public informed as new information becomes available.
Read the full safety alert on the FDA’s website.
The Food and Drug Administration announced a safety alert on Aug. 10, 2017, for liquid-filled intragastric balloon systems, as they have caused five reports of unanticipated deaths that occurred from 2016 to present in patients.
The cause or incidence of patient death is still unknown, and the FDA has not been able to definitively attribute the deaths to the devices or the insertion procedures for these devices. All five reports show that patient deaths occurred within a month or less of balloon placement. In three of the reports, death occurred as soon as 1-3 days after balloon placement. The FDA has also received two additional reports of deaths in the same time period related to potential complications associated with balloon treatment.
The FDA continues to recommend that health care providers closely monitor patients treated with these devices for complications. Any adverse events related to intragastric balloon systems should be reported through MedWatch. The FDA will keep the public informed as new information becomes available.
Read the full safety alert on the FDA’s website.
Painful autoimmune neuropathy may mimic Guillain-Barré syndrome
Patients with an acute transient immune response that is directed against small nerve fibers can display features similar to those of Guillain-Barré syndrome, according to Nobuhiro Yuki, PhD, and associates.
The investigators described three Chinese patients with severe pain in their extremities weeks after infectious illness. Pain greatly improved in two of the patients within days (up to 1 week) of treatment with intravenous immunoglobulin. One patient who refused intravenous immunoglobulin gradually improved with 3 weeks of prednisolone treatment but did not improve as much as the other two.
The investigators found that intrathecal injections of the sera from the acute phase of these patients’ illnesses into nociceptive thermal mouse models induced a transient thermal hypersensitivity, whereas sera from 25 patients with Charcot-Marie-Tooth disease and 25 patients with chronic inflammatory demyelinating polyneuropathy who served as disease controls did not. The intrathecal injections of sera from a healthy donor or from the first two of the three described patients after recovery did not affect thermal sensitivity.
Also, patients’ sera in the acute phase strongly and widely stained small nerve fibers in the dermis of the foot pads of the mice and colocalized with a nerve marker. The sera did not react against myelinated fibers from mouse sciatic nerves. Serum IgG antibodies immunostained the dorsal horn of the lumber spinal cord during the acute phase in all patients, but none of the patients showed reactivity in the convalescent phase.
Patients’ IgG antibodies reacted against surface antigens from the neuron cell body and axon from patients’ sera, but none of the patients with Charcot-Marie-Tooth or chronic inflammatory demyelinating polyneuropathy presented a similar reaction against dorsal root ganglion neurons.
“Our case series and the cases identified in the literature suggest that an acute immune response can be directed against small fibers and exhibit similarities to Guillain-Barré syndrome, including acute preceding infectious illness, a monophasic course, and albuminocytologic dissociation,” the researchers concluded. “It is also worth pointing out that patients with ‘acute small fiber sensory neuropathy’ subsequent to infection or vaccination do not always complain of pain, suggesting a different disease entity.”
Read the full study in Muscle & Nerve (2017. doi: 10.1002/mus.25738).
Patients with an acute transient immune response that is directed against small nerve fibers can display features similar to those of Guillain-Barré syndrome, according to Nobuhiro Yuki, PhD, and associates.
The investigators described three Chinese patients with severe pain in their extremities weeks after infectious illness. Pain greatly improved in two of the patients within days (up to 1 week) of treatment with intravenous immunoglobulin. One patient who refused intravenous immunoglobulin gradually improved with 3 weeks of prednisolone treatment but did not improve as much as the other two.
The investigators found that intrathecal injections of the sera from the acute phase of these patients’ illnesses into nociceptive thermal mouse models induced a transient thermal hypersensitivity, whereas sera from 25 patients with Charcot-Marie-Tooth disease and 25 patients with chronic inflammatory demyelinating polyneuropathy who served as disease controls did not. The intrathecal injections of sera from a healthy donor or from the first two of the three described patients after recovery did not affect thermal sensitivity.
Also, patients’ sera in the acute phase strongly and widely stained small nerve fibers in the dermis of the foot pads of the mice and colocalized with a nerve marker. The sera did not react against myelinated fibers from mouse sciatic nerves. Serum IgG antibodies immunostained the dorsal horn of the lumber spinal cord during the acute phase in all patients, but none of the patients showed reactivity in the convalescent phase.
Patients’ IgG antibodies reacted against surface antigens from the neuron cell body and axon from patients’ sera, but none of the patients with Charcot-Marie-Tooth or chronic inflammatory demyelinating polyneuropathy presented a similar reaction against dorsal root ganglion neurons.
“Our case series and the cases identified in the literature suggest that an acute immune response can be directed against small fibers and exhibit similarities to Guillain-Barré syndrome, including acute preceding infectious illness, a monophasic course, and albuminocytologic dissociation,” the researchers concluded. “It is also worth pointing out that patients with ‘acute small fiber sensory neuropathy’ subsequent to infection or vaccination do not always complain of pain, suggesting a different disease entity.”
Read the full study in Muscle & Nerve (2017. doi: 10.1002/mus.25738).
Patients with an acute transient immune response that is directed against small nerve fibers can display features similar to those of Guillain-Barré syndrome, according to Nobuhiro Yuki, PhD, and associates.
The investigators described three Chinese patients with severe pain in their extremities weeks after infectious illness. Pain greatly improved in two of the patients within days (up to 1 week) of treatment with intravenous immunoglobulin. One patient who refused intravenous immunoglobulin gradually improved with 3 weeks of prednisolone treatment but did not improve as much as the other two.
The investigators found that intrathecal injections of the sera from the acute phase of these patients’ illnesses into nociceptive thermal mouse models induced a transient thermal hypersensitivity, whereas sera from 25 patients with Charcot-Marie-Tooth disease and 25 patients with chronic inflammatory demyelinating polyneuropathy who served as disease controls did not. The intrathecal injections of sera from a healthy donor or from the first two of the three described patients after recovery did not affect thermal sensitivity.
Also, patients’ sera in the acute phase strongly and widely stained small nerve fibers in the dermis of the foot pads of the mice and colocalized with a nerve marker. The sera did not react against myelinated fibers from mouse sciatic nerves. Serum IgG antibodies immunostained the dorsal horn of the lumber spinal cord during the acute phase in all patients, but none of the patients showed reactivity in the convalescent phase.
Patients’ IgG antibodies reacted against surface antigens from the neuron cell body and axon from patients’ sera, but none of the patients with Charcot-Marie-Tooth or chronic inflammatory demyelinating polyneuropathy presented a similar reaction against dorsal root ganglion neurons.
“Our case series and the cases identified in the literature suggest that an acute immune response can be directed against small fibers and exhibit similarities to Guillain-Barré syndrome, including acute preceding infectious illness, a monophasic course, and albuminocytologic dissociation,” the researchers concluded. “It is also worth pointing out that patients with ‘acute small fiber sensory neuropathy’ subsequent to infection or vaccination do not always complain of pain, suggesting a different disease entity.”
Read the full study in Muscle & Nerve (2017. doi: 10.1002/mus.25738).
FROM MUSCLE & NERVE
FDA approves new treatment for adults with HCV
The Food and Drug Administration announced on July 18 the approval of Vosevi to treat adults with chronic hepatitis C virus (HCV) genotypes 1-6 without cirrhosis or with mild cirrhosis.
Vosevi is now the first treatment for patients who have been previously treated with the direct-acting antiviral drug sofosbuvir or other drugs for HCV that inhibit a protein called NS5A. The new drug is a fixed-dose, combination tablet containing sofosbuvir and velpatasvir (both approved before) and a new drug – voxilaprevir.
It is noted that treatment recommendations for Vosevi are different depending on viral genotype and prior treatment history. Vosevi is contraindicated in patients taking the drug rifampin.
“Direct-acting antiviral drugs prevent the virus from multiplying and often cure HCV. Vosevi provides a treatment option for some patients who were not successfully treated with other HCV drugs in the past,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
Read the full press release on the FDA’s website.
The Food and Drug Administration announced on July 18 the approval of Vosevi to treat adults with chronic hepatitis C virus (HCV) genotypes 1-6 without cirrhosis or with mild cirrhosis.
Vosevi is now the first treatment for patients who have been previously treated with the direct-acting antiviral drug sofosbuvir or other drugs for HCV that inhibit a protein called NS5A. The new drug is a fixed-dose, combination tablet containing sofosbuvir and velpatasvir (both approved before) and a new drug – voxilaprevir.
It is noted that treatment recommendations for Vosevi are different depending on viral genotype and prior treatment history. Vosevi is contraindicated in patients taking the drug rifampin.
“Direct-acting antiviral drugs prevent the virus from multiplying and often cure HCV. Vosevi provides a treatment option for some patients who were not successfully treated with other HCV drugs in the past,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
Read the full press release on the FDA’s website.
The Food and Drug Administration announced on July 18 the approval of Vosevi to treat adults with chronic hepatitis C virus (HCV) genotypes 1-6 without cirrhosis or with mild cirrhosis.
Vosevi is now the first treatment for patients who have been previously treated with the direct-acting antiviral drug sofosbuvir or other drugs for HCV that inhibit a protein called NS5A. The new drug is a fixed-dose, combination tablet containing sofosbuvir and velpatasvir (both approved before) and a new drug – voxilaprevir.
It is noted that treatment recommendations for Vosevi are different depending on viral genotype and prior treatment history. Vosevi is contraindicated in patients taking the drug rifampin.
“Direct-acting antiviral drugs prevent the virus from multiplying and often cure HCV. Vosevi provides a treatment option for some patients who were not successfully treated with other HCV drugs in the past,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
Read the full press release on the FDA’s website.
Course and outcome of Guillain-Barré syndrome measured in ongoing study
The International Guillain-Barré Syndrome Outcome Study (IGOS) is actively recruiting patients with Guillain-Barré Syndrome (GBS) to examine disease course and outcome, according to ClinicalTrials.gov.
IGOS is conducted by the members of the Inflammatory Neuropathy Consortium and Peripheral Nerve Society, and the researchers plan to identify clinical and biological determinants and predictors of disease course and outcome in individual patients with GBS as early as possible after onset of disease. It is a prospective study with standardized collection of clinical data and biomaterials from a large group of well-defined GBS patients during a long follow-up period. Patients will be divided into four cohorts: GBS patients with a follow-up of 1-3 years, normal controls, infectious controls, and other neurological diseases.
The primary outcome is to receive a disability score and Medical Research Council sum score within a 1-year time frame. Secondary outcomes include Overall Neuropathy Limitations Scale, Fatigue Severity Scale, EurQol EQ-5D health questionnaire, and Rasch-built Overall Disability Scale, all in a 1-year time frame. The information will be used to understand the diversity in clinical presentation and response to treatment of GBS and will also be used to develop new prognostic models to predict the clinical course and outcome accurately in individual patients with GBS.
Enrollment for the study started May 2012 and the researchers aim to enroll an estimated 4,000 participants. As of April 2017, the IGOS has enrolled more than 1,500 patients, according to the study’s website. The study is expected to be completed by January 2019. All patients with GBS or variants of GBS, including the Miller Fisher syndrome and overlap syndromes, are eligible for the study.
Currently, patients with GBS have not shown improvement over the last 20 years. It is estimated that 10%-20% of patients remain severely disabled and about 5% die from GBS.
Find the full summary here.
The International Guillain-Barré Syndrome Outcome Study (IGOS) is actively recruiting patients with Guillain-Barré Syndrome (GBS) to examine disease course and outcome, according to ClinicalTrials.gov.
IGOS is conducted by the members of the Inflammatory Neuropathy Consortium and Peripheral Nerve Society, and the researchers plan to identify clinical and biological determinants and predictors of disease course and outcome in individual patients with GBS as early as possible after onset of disease. It is a prospective study with standardized collection of clinical data and biomaterials from a large group of well-defined GBS patients during a long follow-up period. Patients will be divided into four cohorts: GBS patients with a follow-up of 1-3 years, normal controls, infectious controls, and other neurological diseases.
The primary outcome is to receive a disability score and Medical Research Council sum score within a 1-year time frame. Secondary outcomes include Overall Neuropathy Limitations Scale, Fatigue Severity Scale, EurQol EQ-5D health questionnaire, and Rasch-built Overall Disability Scale, all in a 1-year time frame. The information will be used to understand the diversity in clinical presentation and response to treatment of GBS and will also be used to develop new prognostic models to predict the clinical course and outcome accurately in individual patients with GBS.
Enrollment for the study started May 2012 and the researchers aim to enroll an estimated 4,000 participants. As of April 2017, the IGOS has enrolled more than 1,500 patients, according to the study’s website. The study is expected to be completed by January 2019. All patients with GBS or variants of GBS, including the Miller Fisher syndrome and overlap syndromes, are eligible for the study.
Currently, patients with GBS have not shown improvement over the last 20 years. It is estimated that 10%-20% of patients remain severely disabled and about 5% die from GBS.
Find the full summary here.
The International Guillain-Barré Syndrome Outcome Study (IGOS) is actively recruiting patients with Guillain-Barré Syndrome (GBS) to examine disease course and outcome, according to ClinicalTrials.gov.
IGOS is conducted by the members of the Inflammatory Neuropathy Consortium and Peripheral Nerve Society, and the researchers plan to identify clinical and biological determinants and predictors of disease course and outcome in individual patients with GBS as early as possible after onset of disease. It is a prospective study with standardized collection of clinical data and biomaterials from a large group of well-defined GBS patients during a long follow-up period. Patients will be divided into four cohorts: GBS patients with a follow-up of 1-3 years, normal controls, infectious controls, and other neurological diseases.
The primary outcome is to receive a disability score and Medical Research Council sum score within a 1-year time frame. Secondary outcomes include Overall Neuropathy Limitations Scale, Fatigue Severity Scale, EurQol EQ-5D health questionnaire, and Rasch-built Overall Disability Scale, all in a 1-year time frame. The information will be used to understand the diversity in clinical presentation and response to treatment of GBS and will also be used to develop new prognostic models to predict the clinical course and outcome accurately in individual patients with GBS.
Enrollment for the study started May 2012 and the researchers aim to enroll an estimated 4,000 participants. As of April 2017, the IGOS has enrolled more than 1,500 patients, according to the study’s website. The study is expected to be completed by January 2019. All patients with GBS or variants of GBS, including the Miller Fisher syndrome and overlap syndromes, are eligible for the study.
Currently, patients with GBS have not shown improvement over the last 20 years. It is estimated that 10%-20% of patients remain severely disabled and about 5% die from GBS.
Find the full summary here.
SUMMARY FROM CLINICALTRIALS.GOV
FDA approves abatacept for adults with psoriatic arthritis
The Food and Drug Administration has approved abatacept, a selective T-cell costimulation modulator, for treating adults with active psoriatic arthritis (PsA), the manufacturer, Bristol-Myers Squibb, has announced.
Approval of abatacept (Orencia) was based on two randomized, double-blind, placebo-controlled studies (PsA-I and PsA-II) in 594 adults with PsA for more than 7 years, according to the July 6 announcement. Patients had active PsA (at least three swollen joints and at least three tender joints), despite previous disease-modifying antirheumatic drug (DMARD) therapy and had one qualifying psoriatic skin lesion measuring at least 2 cm in diameter. The studies included patients treated with TNF inhibitors (TNFi) previously.
In the PsA-II trial, 424 patients received weekly doses of placebo or abatacept 25 mg administered subcutaneously (SC) without a loading dose for 24 weeks, followed by open-label abatacept at a dose of 125 mg SC weekly.
Compared with those on placebo, more patients treated with abatacept 10 mg/kg IV or 125 mg SC achieved an ACR 20 (American College of Rheumatology 20) response at 24 weeks: 47.5% vs. 19.0% and 39.4% vs. 22.3%, respectively (P less than .05).
Other results included a greater proportion of abatacept SC patients with at least a 0.35 decrease from baseline on the Health Assessment Questionnaire-Disability Index: 31% vs. 24% on placebo at 24 weeks. Responses were seen regardless of prior anti-TNFi treatment and regardless of concomitant non-biologic DMARD treatment. In addition, patients on abatacept IV and SC had improvements in enthesitis and dactylitis at 24 weeks.
The safety profile of abatacept in the two studies was “consistent with the safety profile” in rheumatoid arthritis, according to the company release.
Abatacept, initially approved in 2005, was previously approved for RA in adults and for juvenile idiopathic arthritis
Find the updated prescribing information for abatacept here.
The Food and Drug Administration has approved abatacept, a selective T-cell costimulation modulator, for treating adults with active psoriatic arthritis (PsA), the manufacturer, Bristol-Myers Squibb, has announced.
Approval of abatacept (Orencia) was based on two randomized, double-blind, placebo-controlled studies (PsA-I and PsA-II) in 594 adults with PsA for more than 7 years, according to the July 6 announcement. Patients had active PsA (at least three swollen joints and at least three tender joints), despite previous disease-modifying antirheumatic drug (DMARD) therapy and had one qualifying psoriatic skin lesion measuring at least 2 cm in diameter. The studies included patients treated with TNF inhibitors (TNFi) previously.
In the PsA-II trial, 424 patients received weekly doses of placebo or abatacept 25 mg administered subcutaneously (SC) without a loading dose for 24 weeks, followed by open-label abatacept at a dose of 125 mg SC weekly.
Compared with those on placebo, more patients treated with abatacept 10 mg/kg IV or 125 mg SC achieved an ACR 20 (American College of Rheumatology 20) response at 24 weeks: 47.5% vs. 19.0% and 39.4% vs. 22.3%, respectively (P less than .05).
Other results included a greater proportion of abatacept SC patients with at least a 0.35 decrease from baseline on the Health Assessment Questionnaire-Disability Index: 31% vs. 24% on placebo at 24 weeks. Responses were seen regardless of prior anti-TNFi treatment and regardless of concomitant non-biologic DMARD treatment. In addition, patients on abatacept IV and SC had improvements in enthesitis and dactylitis at 24 weeks.
The safety profile of abatacept in the two studies was “consistent with the safety profile” in rheumatoid arthritis, according to the company release.
Abatacept, initially approved in 2005, was previously approved for RA in adults and for juvenile idiopathic arthritis
Find the updated prescribing information for abatacept here.
The Food and Drug Administration has approved abatacept, a selective T-cell costimulation modulator, for treating adults with active psoriatic arthritis (PsA), the manufacturer, Bristol-Myers Squibb, has announced.
Approval of abatacept (Orencia) was based on two randomized, double-blind, placebo-controlled studies (PsA-I and PsA-II) in 594 adults with PsA for more than 7 years, according to the July 6 announcement. Patients had active PsA (at least three swollen joints and at least three tender joints), despite previous disease-modifying antirheumatic drug (DMARD) therapy and had one qualifying psoriatic skin lesion measuring at least 2 cm in diameter. The studies included patients treated with TNF inhibitors (TNFi) previously.
In the PsA-II trial, 424 patients received weekly doses of placebo or abatacept 25 mg administered subcutaneously (SC) without a loading dose for 24 weeks, followed by open-label abatacept at a dose of 125 mg SC weekly.
Compared with those on placebo, more patients treated with abatacept 10 mg/kg IV or 125 mg SC achieved an ACR 20 (American College of Rheumatology 20) response at 24 weeks: 47.5% vs. 19.0% and 39.4% vs. 22.3%, respectively (P less than .05).
Other results included a greater proportion of abatacept SC patients with at least a 0.35 decrease from baseline on the Health Assessment Questionnaire-Disability Index: 31% vs. 24% on placebo at 24 weeks. Responses were seen regardless of prior anti-TNFi treatment and regardless of concomitant non-biologic DMARD treatment. In addition, patients on abatacept IV and SC had improvements in enthesitis and dactylitis at 24 weeks.
The safety profile of abatacept in the two studies was “consistent with the safety profile” in rheumatoid arthritis, according to the company release.
Abatacept, initially approved in 2005, was previously approved for RA in adults and for juvenile idiopathic arthritis
Find the updated prescribing information for abatacept here.
Cotempla XR-ODT approved for children, adolescents with ADHD
The Food and Drug Administration has approved the first methylphenidate extended-release orally disintegrating tablet for treating ADHD in patients aged 6-17 years old, Neos Therapeutics announced June 19.
The company said the approval came after a phase III trial showed that treatment in a laboratory classroom with the drug, called Cotempla XR-ODT, showed a significant improvement in attention-deficit/hyperactivity disorder symptom control when compared with a placebo across the classroom day (placebo-subtracted difference of –11). The onset of effect was shown at 1 hour post-dose and lasted through 12 hours. No serious adverse events were reported during the trial, and the adverse event profile was consistent with the established safety profile for other extended-release methylphenidate products.
Cotempla XR-ODT will be available commercially in a portable, child-resistant blister pack in the fall of 2017.
Find the full press release on Neos Therapeutics website.
The Food and Drug Administration has approved the first methylphenidate extended-release orally disintegrating tablet for treating ADHD in patients aged 6-17 years old, Neos Therapeutics announced June 19.
The company said the approval came after a phase III trial showed that treatment in a laboratory classroom with the drug, called Cotempla XR-ODT, showed a significant improvement in attention-deficit/hyperactivity disorder symptom control when compared with a placebo across the classroom day (placebo-subtracted difference of –11). The onset of effect was shown at 1 hour post-dose and lasted through 12 hours. No serious adverse events were reported during the trial, and the adverse event profile was consistent with the established safety profile for other extended-release methylphenidate products.
Cotempla XR-ODT will be available commercially in a portable, child-resistant blister pack in the fall of 2017.
Find the full press release on Neos Therapeutics website.
The Food and Drug Administration has approved the first methylphenidate extended-release orally disintegrating tablet for treating ADHD in patients aged 6-17 years old, Neos Therapeutics announced June 19.
The company said the approval came after a phase III trial showed that treatment in a laboratory classroom with the drug, called Cotempla XR-ODT, showed a significant improvement in attention-deficit/hyperactivity disorder symptom control when compared with a placebo across the classroom day (placebo-subtracted difference of –11). The onset of effect was shown at 1 hour post-dose and lasted through 12 hours. No serious adverse events were reported during the trial, and the adverse event profile was consistent with the established safety profile for other extended-release methylphenidate products.
Cotempla XR-ODT will be available commercially in a portable, child-resistant blister pack in the fall of 2017.
Find the full press release on Neos Therapeutics website.
Mydayis approved for teens, adults with ADHD
The Food and Drug Administration has approved a once-a-day treatment for patients aged 13 years and older with ADHD, Shire announced June 20 in a press release.
The approval of Mydayis was based on results from 16 clinical studies evaluating the medication in more than 1,600 adolescents (aged 13-17 years) and adults with attention-deficit/hyperactivity disorder. In the placebo-controlled clinical studies, Mydayis significantly improved symptoms of ADHD, as measured by the ADHD-RS-IV and the Permanent Product Measure of Performance (PERMP), in adults and adolescents. Improvement on the PERMP reached statistical significance beginning at 2 or 4 hours post dose and lasting up to 16 hours post dose.
The medication, an amphetamine product, consists of three different types of drug-releasing beads.
“With this approval, we hope to help patients who need a once-daily treatment option,” Flemming Ornskov, MD, MPH, said in a press release. Dr. Ornskov is CEO of Shire.
It is estimated that 4.4% of adults have ADHD in the United States, and 50%-66% of children with ADHD may continue to have symptoms of the disorder as adults.
Mydayis will be commercially available in the United States in the third quarter of 2017.
Read the full press release here.
The Food and Drug Administration has approved a once-a-day treatment for patients aged 13 years and older with ADHD, Shire announced June 20 in a press release.
The approval of Mydayis was based on results from 16 clinical studies evaluating the medication in more than 1,600 adolescents (aged 13-17 years) and adults with attention-deficit/hyperactivity disorder. In the placebo-controlled clinical studies, Mydayis significantly improved symptoms of ADHD, as measured by the ADHD-RS-IV and the Permanent Product Measure of Performance (PERMP), in adults and adolescents. Improvement on the PERMP reached statistical significance beginning at 2 or 4 hours post dose and lasting up to 16 hours post dose.
The medication, an amphetamine product, consists of three different types of drug-releasing beads.
“With this approval, we hope to help patients who need a once-daily treatment option,” Flemming Ornskov, MD, MPH, said in a press release. Dr. Ornskov is CEO of Shire.
It is estimated that 4.4% of adults have ADHD in the United States, and 50%-66% of children with ADHD may continue to have symptoms of the disorder as adults.
Mydayis will be commercially available in the United States in the third quarter of 2017.
Read the full press release here.
The Food and Drug Administration has approved a once-a-day treatment for patients aged 13 years and older with ADHD, Shire announced June 20 in a press release.
The approval of Mydayis was based on results from 16 clinical studies evaluating the medication in more than 1,600 adolescents (aged 13-17 years) and adults with attention-deficit/hyperactivity disorder. In the placebo-controlled clinical studies, Mydayis significantly improved symptoms of ADHD, as measured by the ADHD-RS-IV and the Permanent Product Measure of Performance (PERMP), in adults and adolescents. Improvement on the PERMP reached statistical significance beginning at 2 or 4 hours post dose and lasting up to 16 hours post dose.
The medication, an amphetamine product, consists of three different types of drug-releasing beads.
“With this approval, we hope to help patients who need a once-daily treatment option,” Flemming Ornskov, MD, MPH, said in a press release. Dr. Ornskov is CEO of Shire.
It is estimated that 4.4% of adults have ADHD in the United States, and 50%-66% of children with ADHD may continue to have symptoms of the disorder as adults.
Mydayis will be commercially available in the United States in the third quarter of 2017.
Read the full press release here.
PBC incidence remains stable in rural parts of U.S.
The incidence of primary biliary cholangitis (PBC) in rural parts of the midwestern United States has remained stable over the last 2 decades, allowing better prognosis and improved survival rates.
In a population-based cohort study, Rajan Kanth, MD, and his associates researched 79 incident PBC cases observed in the Marshfield Epidemiologic Study Area (MESA) of 24 zip codes in central and northern Wisconsin between 1992 and 2011. The overall age- and sex-standardized PBC incidence rate was 4.9 cases per 100,000 person-years. The annual incidence rate of PBC increased; however, it was not significant (P = .114) during the 20-year study time frame. In women, PBC ranged from a low of 6.9 cases per 100,000 person-years in 1992-1996 to a high of 11.3 cases per 100,000 person-years in 2002-2006. The sex-specific comparisons were not significant at any time during the 5-year period.
After a mean follow-up of 7.3 years, 23 (29%) patients with PBC died. The estimated 10-year survival of PBC cases in MESA was 76%.
Researchers noted the MESA source population grew over the 20-year study time frame, going from a low of 364,722 MESA person-years in 1992-1996 to a high of 409,670 person-years in 2007-2011. The proportion of men and women in MESA were consistent throughout the study, but there was a general population aging trend with a 29% increase in the number of individuals aged 40-69 years in 2007-2011 relative to 1992-1996.
“The overall incidence of PBC in a Midwestern population of the United States has remained relatively stable over the last two decades,” researchers concluded. “Results suggest that the overall incidence of PBC in the United States is not rising quickly, and that patients with PBC have generally improved prognosis and survival.”
Find the full study in Clinical Medicine & Research (2017. doi: 10.3121/cmr.2017.1351).
The incidence of primary biliary cholangitis (PBC) in rural parts of the midwestern United States has remained stable over the last 2 decades, allowing better prognosis and improved survival rates.
In a population-based cohort study, Rajan Kanth, MD, and his associates researched 79 incident PBC cases observed in the Marshfield Epidemiologic Study Area (MESA) of 24 zip codes in central and northern Wisconsin between 1992 and 2011. The overall age- and sex-standardized PBC incidence rate was 4.9 cases per 100,000 person-years. The annual incidence rate of PBC increased; however, it was not significant (P = .114) during the 20-year study time frame. In women, PBC ranged from a low of 6.9 cases per 100,000 person-years in 1992-1996 to a high of 11.3 cases per 100,000 person-years in 2002-2006. The sex-specific comparisons were not significant at any time during the 5-year period.
After a mean follow-up of 7.3 years, 23 (29%) patients with PBC died. The estimated 10-year survival of PBC cases in MESA was 76%.
Researchers noted the MESA source population grew over the 20-year study time frame, going from a low of 364,722 MESA person-years in 1992-1996 to a high of 409,670 person-years in 2007-2011. The proportion of men and women in MESA were consistent throughout the study, but there was a general population aging trend with a 29% increase in the number of individuals aged 40-69 years in 2007-2011 relative to 1992-1996.
“The overall incidence of PBC in a Midwestern population of the United States has remained relatively stable over the last two decades,” researchers concluded. “Results suggest that the overall incidence of PBC in the United States is not rising quickly, and that patients with PBC have generally improved prognosis and survival.”
Find the full study in Clinical Medicine & Research (2017. doi: 10.3121/cmr.2017.1351).
The incidence of primary biliary cholangitis (PBC) in rural parts of the midwestern United States has remained stable over the last 2 decades, allowing better prognosis and improved survival rates.
In a population-based cohort study, Rajan Kanth, MD, and his associates researched 79 incident PBC cases observed in the Marshfield Epidemiologic Study Area (MESA) of 24 zip codes in central and northern Wisconsin between 1992 and 2011. The overall age- and sex-standardized PBC incidence rate was 4.9 cases per 100,000 person-years. The annual incidence rate of PBC increased; however, it was not significant (P = .114) during the 20-year study time frame. In women, PBC ranged from a low of 6.9 cases per 100,000 person-years in 1992-1996 to a high of 11.3 cases per 100,000 person-years in 2002-2006. The sex-specific comparisons were not significant at any time during the 5-year period.
After a mean follow-up of 7.3 years, 23 (29%) patients with PBC died. The estimated 10-year survival of PBC cases in MESA was 76%.
Researchers noted the MESA source population grew over the 20-year study time frame, going from a low of 364,722 MESA person-years in 1992-1996 to a high of 409,670 person-years in 2007-2011. The proportion of men and women in MESA were consistent throughout the study, but there was a general population aging trend with a 29% increase in the number of individuals aged 40-69 years in 2007-2011 relative to 1992-1996.
“The overall incidence of PBC in a Midwestern population of the United States has remained relatively stable over the last two decades,” researchers concluded. “Results suggest that the overall incidence of PBC in the United States is not rising quickly, and that patients with PBC have generally improved prognosis and survival.”
Find the full study in Clinical Medicine & Research (2017. doi: 10.3121/cmr.2017.1351).
FROM CLINICAL MEDICINE & RESEARCH
FDA approves Sapien 3 transcatheter valve for bioprosthetic valve failure
The Food and Drug Administration announced June 5 the approval of an expanded indication for the Sapien 3 Transcatheter Heart Valve (THV) for patients with symptomatic heart disease caused by failure of a previously placed bioprosthetic aortic or mitral valve who have a risk of death or severe complications from repeat surgery.
This is the first FDA approval for the expanded use of the Sapien 3 THV as a valve-in-valve treatment. Such procedures provide an alternative to repeat surgery.
“For the first time, a regulatory agency is approving a transcatheter heart valve as a valve-in-valve treatment when bioprosthetic mitral or aortic valves fail in patients who are at high or greater risk of complications from repeat surgery,” Bram Zuckerman, MD, director of the division of cardiovascular devices at the FDA’s Center for Devices and Radiological Health, said in a press release. “This new approval offers U.S. patients with failing surgical bioprosthetic aortic or mitral valves a less-invasive treatment option.”
Originally, the FDA approved the Sapien 3 THV for transcatheter aortic valve replacement (TAVR) as an alternative to surgical aortic valve replacement for patients with native aortic stenosis whose risk for death or severe complications from surgery is high or greater. Then in 2016, the FDA expanded the TAVR indication for Sapien 3 THV to include patients who are at intermediate surgical risk for death or complications.
Read the full press release on the FDA’s website.
The Food and Drug Administration announced June 5 the approval of an expanded indication for the Sapien 3 Transcatheter Heart Valve (THV) for patients with symptomatic heart disease caused by failure of a previously placed bioprosthetic aortic or mitral valve who have a risk of death or severe complications from repeat surgery.
This is the first FDA approval for the expanded use of the Sapien 3 THV as a valve-in-valve treatment. Such procedures provide an alternative to repeat surgery.
“For the first time, a regulatory agency is approving a transcatheter heart valve as a valve-in-valve treatment when bioprosthetic mitral or aortic valves fail in patients who are at high or greater risk of complications from repeat surgery,” Bram Zuckerman, MD, director of the division of cardiovascular devices at the FDA’s Center for Devices and Radiological Health, said in a press release. “This new approval offers U.S. patients with failing surgical bioprosthetic aortic or mitral valves a less-invasive treatment option.”
Originally, the FDA approved the Sapien 3 THV for transcatheter aortic valve replacement (TAVR) as an alternative to surgical aortic valve replacement for patients with native aortic stenosis whose risk for death or severe complications from surgery is high or greater. Then in 2016, the FDA expanded the TAVR indication for Sapien 3 THV to include patients who are at intermediate surgical risk for death or complications.
Read the full press release on the FDA’s website.
The Food and Drug Administration announced June 5 the approval of an expanded indication for the Sapien 3 Transcatheter Heart Valve (THV) for patients with symptomatic heart disease caused by failure of a previously placed bioprosthetic aortic or mitral valve who have a risk of death or severe complications from repeat surgery.
This is the first FDA approval for the expanded use of the Sapien 3 THV as a valve-in-valve treatment. Such procedures provide an alternative to repeat surgery.
“For the first time, a regulatory agency is approving a transcatheter heart valve as a valve-in-valve treatment when bioprosthetic mitral or aortic valves fail in patients who are at high or greater risk of complications from repeat surgery,” Bram Zuckerman, MD, director of the division of cardiovascular devices at the FDA’s Center for Devices and Radiological Health, said in a press release. “This new approval offers U.S. patients with failing surgical bioprosthetic aortic or mitral valves a less-invasive treatment option.”
Originally, the FDA approved the Sapien 3 THV for transcatheter aortic valve replacement (TAVR) as an alternative to surgical aortic valve replacement for patients with native aortic stenosis whose risk for death or severe complications from surgery is high or greater. Then in 2016, the FDA expanded the TAVR indication for Sapien 3 THV to include patients who are at intermediate surgical risk for death or complications.
Read the full press release on the FDA’s website.
Parkinsonian symptoms at diagnosis raise synucleinopathy mortality risk
The elevated risk of death for patients with clinically diagnosed synucleinopathies and symptoms of parkinsonism is highest for those with multiple system atrophy with predominant parkinsonism (MSA-p), followed by dementia with Lewy bodies (DLB), Parkinson’s disease dementia (PDD), and Parkinson’s disease (PD), according to Rodolfo Savica, MD, PhD, and his associates.
The investigators compared 461 patients who had onset of a clinically presumed synucleinopathy manifesting as parkinsonism from 1991 to 2010 with 452 age- and sex-matched referent participants from the general population who were free of parkinsonism and tremor of any type in the year of onset of the other patients’ synucleinopathies. Of the 461 patients with the presumed synucleinopathies, 316 (68.6%) died during follow-up and 311 had a known cause of death (98.4%). Of the 452 referent participants, 220 (48.7%) died during follow-up and 216 had a known cause of death (98.2%). The highest risk of death was among patients with MSA-p (hazard ratio, 10.51) when compared with referent participants. The remaining patients also had elevated risk of death: DLB (HR, 3.94), PDD (HR, 3.86), and PD (HR, 1.75).
Neurodegenerative disease was the most frequent cause of death among patients for all synucleinopathies (31.5%) and in PD alone (25.6%), and cardiovascular events were the second most common cause of death (15.7%). Among the referent participants, cardiovascular events were the most common cause of death (25.5%).
The results were consistent with the causes of death observed among patients with DLB, PDD, and MSA-p; however, the researchers said the sample size was too limited to observe a sufficient number of events. They also noted that there was no significant interaction with sex and age in predicting survival rates for any type of synucleinopathy.
“Our findings contribute important new evidence about the natural history and survival of people affected by synucleinopathies of various types,” the researchers concluded. “Our results may be helpful to guide clinicians counseling patients and caregivers.”
Find the full study in JAMA Neurology (doi: 10.1001/jamaneurol.2017.0603).
The elevated risk of death for patients with clinically diagnosed synucleinopathies and symptoms of parkinsonism is highest for those with multiple system atrophy with predominant parkinsonism (MSA-p), followed by dementia with Lewy bodies (DLB), Parkinson’s disease dementia (PDD), and Parkinson’s disease (PD), according to Rodolfo Savica, MD, PhD, and his associates.
The investigators compared 461 patients who had onset of a clinically presumed synucleinopathy manifesting as parkinsonism from 1991 to 2010 with 452 age- and sex-matched referent participants from the general population who were free of parkinsonism and tremor of any type in the year of onset of the other patients’ synucleinopathies. Of the 461 patients with the presumed synucleinopathies, 316 (68.6%) died during follow-up and 311 had a known cause of death (98.4%). Of the 452 referent participants, 220 (48.7%) died during follow-up and 216 had a known cause of death (98.2%). The highest risk of death was among patients with MSA-p (hazard ratio, 10.51) when compared with referent participants. The remaining patients also had elevated risk of death: DLB (HR, 3.94), PDD (HR, 3.86), and PD (HR, 1.75).
Neurodegenerative disease was the most frequent cause of death among patients for all synucleinopathies (31.5%) and in PD alone (25.6%), and cardiovascular events were the second most common cause of death (15.7%). Among the referent participants, cardiovascular events were the most common cause of death (25.5%).
The results were consistent with the causes of death observed among patients with DLB, PDD, and MSA-p; however, the researchers said the sample size was too limited to observe a sufficient number of events. They also noted that there was no significant interaction with sex and age in predicting survival rates for any type of synucleinopathy.
“Our findings contribute important new evidence about the natural history and survival of people affected by synucleinopathies of various types,” the researchers concluded. “Our results may be helpful to guide clinicians counseling patients and caregivers.”
Find the full study in JAMA Neurology (doi: 10.1001/jamaneurol.2017.0603).
The elevated risk of death for patients with clinically diagnosed synucleinopathies and symptoms of parkinsonism is highest for those with multiple system atrophy with predominant parkinsonism (MSA-p), followed by dementia with Lewy bodies (DLB), Parkinson’s disease dementia (PDD), and Parkinson’s disease (PD), according to Rodolfo Savica, MD, PhD, and his associates.
The investigators compared 461 patients who had onset of a clinically presumed synucleinopathy manifesting as parkinsonism from 1991 to 2010 with 452 age- and sex-matched referent participants from the general population who were free of parkinsonism and tremor of any type in the year of onset of the other patients’ synucleinopathies. Of the 461 patients with the presumed synucleinopathies, 316 (68.6%) died during follow-up and 311 had a known cause of death (98.4%). Of the 452 referent participants, 220 (48.7%) died during follow-up and 216 had a known cause of death (98.2%). The highest risk of death was among patients with MSA-p (hazard ratio, 10.51) when compared with referent participants. The remaining patients also had elevated risk of death: DLB (HR, 3.94), PDD (HR, 3.86), and PD (HR, 1.75).
Neurodegenerative disease was the most frequent cause of death among patients for all synucleinopathies (31.5%) and in PD alone (25.6%), and cardiovascular events were the second most common cause of death (15.7%). Among the referent participants, cardiovascular events were the most common cause of death (25.5%).
The results were consistent with the causes of death observed among patients with DLB, PDD, and MSA-p; however, the researchers said the sample size was too limited to observe a sufficient number of events. They also noted that there was no significant interaction with sex and age in predicting survival rates for any type of synucleinopathy.
“Our findings contribute important new evidence about the natural history and survival of people affected by synucleinopathies of various types,” the researchers concluded. “Our results may be helpful to guide clinicians counseling patients and caregivers.”
Find the full study in JAMA Neurology (doi: 10.1001/jamaneurol.2017.0603).
FROM JAMA NEUROLOGY