CPAP Not Suggested for Elders, sNDA Accepted for Risdiplam in Presymptomatic SMA, Phase 2 Trial of GV-971 Gets Greenlight

Neurology News Network for the week ending January 29, 2022.

WATCH TIME: 4 minutes

This week Neurology News Network covered an analysis that found limited benefits for elders with obstructive sleep apnea who are treated with CPAP, the FDA's decision on the sNDA of risdiplam for presymptomatic SMA, and the phase 2 trial of sodium oligomannate in patients with Parkinson disease.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

Post hoc data from 2 open-label, multicenter clinical trials showed that individuals with obstructive sleep apnea (OSA) aged at least 70 years old had low adherence to continuous positive airway pressure (CPAP) with limited treatment effects observed. Investigators concluded that the findings do not support the use of CPAP in this age population.At follow-up, investigators recorded a significant improvement in snoring and witnessed apneas with treatment of CPAP, but no significant changes occurred in symptoms such as choking nocturia, or nightmares. Additionally, these patients demonstrated ESS values of 9.1, a decrease of 1.2 points, after adjusting for baseline ESS values, compared to the control group. No significant correlation was observed between the change in ESS values and the number of hours of CPAP use. Quality of life, measured using Quebec Sleep Questionnaire (QSQ), indicated no statistical or clinical improvements for those on CPAP compared with the control group, while adjusting for their corresponding baseline values. Moreover, no evidence of a significant correlation emerged for the changes between groups in the different domains of QSQ and the number of hours of CPAP use.

A supplemental new drug application (sNDA) for risdiplam (Evrysdi; PTC Therapeutics) has been granted priority review by the FDA for treatment of presymptomatic babies under 2 months of age with spinal muscular atrophy (SMA). The treatment is currently used to treat SMA in adults and children 2 months of age and older, and if approved, the sNDA will make risdiplam the first medicine administered at home for presymptomatic babies with SMA.The submission incorporates interim data from the RAINBOWFISH study (NCT03779334), which suggest that following 12 months of treatment with risdiplam, a majority of presymptomatic babies met key milestones of healthy babies, including sitting, standing, and walking, in addition to maintaining the ability to swallow. he RAINBOWFISH study included 5 babies, all of whom maintained the ability to swallow and feed exclusively orally after 12 months of treatment. Additionally, after 12 months, 4 out of 5 babies (80%) receiving risdiplam were able to stand and walk independently, in accordance with the established windows for health children from the World Health Organization.

The FDA has accepted the investigational new drug (IND) application from Green Valley Pharmaceuticals’ and has allowed the company to go ahead with its phase 2 trial assessing sodium oligomannate (GV-971), an agent approved in China for Alzheimer disease (AD), in a global cohort of patients with early-stage Parkinson disease (PD).This multicenter trial will include approximately 300 patients with early-stage PD who will be randomized to either sodium oligomannate or placebo for a 36-week period. Following that, patients may enter into a nonrandomized, 36-week, open-label extension for further testing. No information was provided as to when the study would begin.Although the pathogenesis of PD is not entirely known, research has pointed to its associations with alpha-synuclein aggregation, neuroinflammation, oxidative stress, and mitochondrial dysfunction. It is currently the second most common neurodegenerative disease behind AD, which has also had research that has shown a link between gut inflammation and disease progression.

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