Risdiplam PDUFA Pushed Back, Masitinib Gains NDA, COVID19 Affects CNS

April 11, 2020

Neurology News Network for the week ending April 11, 2020.

This week Neurology News Network discussed the pushed back PDUFA date for the spinal muscular atrophy treatment, risdiplam, as well as the new drug application for masitinib and the first reported central nervous system complication affected by COVID19.

Marco Meglio: 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.

The regulatory decision for the investigational spinal muscular atrophy treatment risdiplam has been pushed back by several months as the FDA reviews new data, according to an announcement from Roche. The drug, an orally active SMN2 splicing modifier potentially effective for the treatment of all types of SMA, had an original PDUFA date of May 24, 2020. The new PDUFA date is now August 24, 2020. The delay is the result of Roche submitting additional data from the pivotal SUNFISH part 2 trial, which demonstrated the drug’s efficacy in patients aged 2 to 25 with SMA types 2 or 3. Stuart Peltz, CEO of PTC Therapeutics said, "We are encouraged that the FDA has no substantive review issues. Their interest in the additional results from the clinical studies demonstrating risdiplam's activity supports our goal of enabling access to this important therapy for all SMA patients,"

AB Science announced that the FDA has accepted its investigational new drug application for masitinib, and thus will proceed with a phase 3 study to test the agent in patients with ALS. Study AB19001 is intended to confirm results from the first phase 2b/3 AB10015 study which demonstrated that masitinib, at 4.5 mg/kg per day in combination with riluzole, significantly slowed decline as measured by the Amytrophic Lateral Sclerosis Functional Rating Scale-revised. Now, AB19001 will enroll 495 patients who will be randomized to 1 of 3 treatment groups in a 1:1:1 ratio. Alan Moussy, co-founder and chief executive officer, AB Science, said in a statement, “That is really good news for all of us and in particular for the patients. We aim to initiate the phase 3 confirmatory trial in ALS as soon as conditions at US clinical sites stabilize post-the coronavirus pandemic.”

As the COVID-19 pandemic rolls on, reports of central nervous system involvement are beginning to emerge. While most evidence remains anecdotal, the reported first case of a rare neurologic complication has been published in Radiology journal. Published images capture the occurrence of acute necrotizing encephalopathy, or ANE, in a female airline employee in her 50s with a confirmed diagnosis of COVID-19. The patient was diagnosed and treated at Henry Ford Hospital System in Detroit, Michigan, where she presented with a 3-day history of fever, cough, and altered mental status. The case authors reported that the infection contributed to cytokine storm syndrome. As more patients are diagnosed with COVID-19, the researchers wrote, it’s likely that more will also be diagnosed with ANE.

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