Pimavanserin Gets NDA Nod, Ubrogepant Improves Chronic Migraine, Risdiplam Increases Motor Function

This week Neurology News Network details the new drug application additional indication of Parkinson disease treatment pimavanserin, as well as the results of a study of ubrogepant in episodic and chronic migraine, and additional data on results of the SUNFISH and JEWELFISH trials of risdiplam in patients with spinal muscular atrophy.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

A supplemental new drug application has been submitted to the FDA for an additional indication for pimavanserin, this time for the treatment of delusions and hallucinations associated with dementia-related psychosis. The Acadia Pharmaceuticals drug was previously approved in 2016 for a similar indication in Parkinson disease psychosis — the first therapy to address this debilitating symptom of the movement disorder. “This is an important step forward for the approximately 2.4 million people in the US who suffer from dementia-related hallucinations and delusions, representing a large unmet need with currently no approved treatment options,” said Steve Davis, Acadia’s chief executive order, in a statement. If it's approved, this will be the first drug for any neuropsychiatric syndrome in any dementia disorder.

Preliminary results from a real-world study evaluating ubrogepant, lasmiditan, and rimegepant in patients with chronic and episodic migraine revealed that ubrogepant showed significant efficacy with regard to pain freedom and headache relief at 2 hours post-dose. The data, which at time of presentation only included survey results for ubrogepant, showed that 39 of the 57 (68%) respondents reported that ubrogepant had completely aborted headache pain in 2 hours. Among the 57 respondents, 81% reported suffering from chronic migraine. The survery results for rimgepant and lasmiditan are still under analysis. Ubrogepant was approved for the acute treatment of migraine with or without aura by the FDA in December 2019, making it the first-in-class oral CGRP antagonist for this indication.

Two-year data from Part 1 of the SUNFISH trial and 12-month data from the JEWELFISH trial of risdiplam demonstrated the treatment’s ability to significantly improve motor function after 24 months as well as showing rapid and sustained increases in survival motor neuron protein levels in those with spinal muscular atrophy. Results of the 2 studies, presented at this year’s Cure SMA Annual Conference, also showed no new safety signals, with an overall adverse event profile consistent with that of treatment-naïve patients. Exploratory efficacy analysis of Part 1 data from SUNFISH focused on motor function using the Motor Function Measure scale. In a weighted analysis comparing the data with robust natural history comparator cohort, MFM total change from baseline at 24 months was greater in patients who received risdiplam.

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