CMS Adds Coverage for Antiamyloid Therapies, NDA for Migraine Agent Accepted, INTUNE Study Ahead of Schedule

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

According to a new announcement, the Centers for Medicare & Medicaid Services (CMS) plans to provide coverage for therapies designed to slow the progression of Alzheimer disease (AD) that have received traditional approval. The announcement comes months after the agency doubled down on its April 2022 decision to restrict the coverage of FDA-approved antiamyloid therapies to treat the neurodegenerative disease. Earlier this year, in January, the FDA approved lecanemab (Leqembi; Eisai) for the treatment of patients with early-stage AD under the accelerated approval pathway, joining aducanumab (Aduhelm; Biogen) as the only antiamyloid therapies in its class. Shortly after, Eisai filed for traditional approval of the agent, using the phase 3 Clarity AD study as the confirmatory study.2 A few weeks before its expected PDUFA date of July 6, 2023, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will discuss the results of the trial to see if they warrant a traditional approval. That meeting is slated for June 9, 2023.

The FDA has accepted Satsuma Pharmaceuticals’ 505(b)(2) new drug application (NDA) for its novel, investigational DHE nasal powder product, STS101, for the acute treatment of migraine. The agency is expected to make a decision on the therapy by January 2024. The NDA was backed by findings from a phase 1 comparative pharmacokinetic and safety trial and the phase 3, long-term ASCEND trail. Based on previous communications with the FDA, results from the phase 3 SUMMIT trial may be included in the NDA, although they are not required for approval. STS101 is designed to provide significant benefits vs existing acute treatments for migraine, including the combination of quick and convenient self-administration and other clinical advantages that current DHE liquid nasal spray products and injectable dosage forms lack.

Harmony Biosciences announced that enrollment for the phase 3 INTUNE study assessing pitolisant (Wakix) in patients with idiopathic hypersomnia is 9 months ahead of schedule, with topline findings anticipated to be reported in the fourth quarter of 2023. INTUNE, a double-blind, placebo-controlled trail spanning 58 clinical sites in the US, assesses the efficacy and safety of pitolisant in 200 adults with IH, a condition traditionally treated with off-label, wake-promoting products. Announced in April 2022, the trial consists of a screening period, an 8-week open-label phase, and a 4-week double-blind randomized withdrawal phase. Pitolisant is a novel selective histamine 3(H3) receptor antagonist/inverse agonist that increases the synthesis and release of wake-promoting histamine via a once-daily, oral administration. It remains the only FDA-approved treatment for both excessive daytime sleepiness (EDS) and cataplexy in narcolepsy that is not a controlled substance.

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