sNDA Accepted For Rimegepant, FDA Expands Pitolisant Indication, Givinostat Wins Rare Pediatric Disease Designation


Neurology News Network for the week ending October 17, 2020.

This week Neurology News Network covered the supplemental new drug application of rimegepant, the expanded indication for pitolisant for the treatment of cataplexy, and the rare pediatric disease designation granted to givinostat.

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.

Biohaven Pharmaceuticals has announced that its supplemental new drug application for rimegepant (Nurtec ODT) in the preventive treatment of migraine has been accepted by the FDA, with a planned Prescription Drug User Fee Act action date set for the second quarter of 2021. The orally disintegrating anti-calcitonin gene-related peptide (CGRP) tablet was originally approved in a 75-mg dose for the acute treatment of migraine in February 2020, marking the first approval for Biohaven.If approved for this second indication, rimegepant will be the first and only CGRP-targeting therapy with indications for both preventive and acute treatment of migraine.The basis of this new sNDA is built on data from the company’s pivotal study—known as Study 305—in migraine prevention, which showed the same dosage was associated with a significant 4.3-day reduction in monthly migraine days.

Harmony Biosciences has announced that the FDA has expanded the indication for pitolisant for the treatment of cataplexy in adults with narcolepsy. Notably, pitolisant is the only FDA-approved treatment for both excessive daytime sleepiness and cataplexy in narcolepsy that is not a scheduled controlled substance.Pitolisant is a novel, selective histamine 3 receptor antagonist/inverse agonist that increases the synthesis and release of wake-promoting histamine via a once-daily, oral administration. The expanded approval was based on results from the HARMONY CTP and HARMONY 1 randomized, controlled clinical trials, the results of which were reanalyzed after the FDA issued Harmony a complete response letter in August 2019 regarding data supporting the efficacy of pitolisant for the reduction of cataplexy. Results from the CTP study demonstrated a 75% reduction in weekly cataplexy rate in patients taking pitolisant compared with 38% in patients in the placebo group.

The FDA has granted rare pediatric disease designation to givinostat, a histone deacetylase (HDAC) inhibitor, for the treatment of Duchenne muscular dystrophy. The investigational drug is the subject of the phase 3 EPIDYS clinical trial, which completed enrollment at the end of September and remains on track to read out topline data in June 2022. Notably, the last patient is expected to complete the 72-week treatment period in the first quarter of 2022. Paolo Bettica, MD, PhD, chief medical officer, Italfarmaco Group, said in a statement, "This is the third regulatory designation we have received from the FDA for givinostat after the orphan drug designation and fast track designation and reflects the agency’s recognition of givinostat’s potential to treat DMD, a devastating genetic disease.”

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