A Pivotal FDA Review and Potential Commercial Opportunity

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A Pivotal FDA Review and Potential Commercial Opportunity

A Closer Look

The FDA is scheduled to meet with the Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2nd to review Zevra Therapeutics Inc.'s marketing application for ZVRA arimoclomol, a potential treatment for Niemann-Pick disease type C (NPC). This follows a complete response letter issued in 2021, requesting additional evidence to support the validity of the 5-domain NPC Clinical Severity Scale.

Analysts at William Blair believe the data supports arimoclomol's efficacy and safety, and anticipate strong patient advocacy at the meeting. Unlike the previous filing, patient groups have already submitted letters of support, including a petition with nearly 1,000 signatures.

If approved, arimoclomol would represent a significant commercial opportunity for Zevra, despite the ultra-rare nature of NPC. The company estimates around 900 patients in the U.S., with 300-400 currently diagnosed. Zevra has established a commercial infrastructure for Olpruva, a similar drug, and expects a strong launch for arimoclomol, leveraging existing early access program participants and attracting new patients.

Pricing will be a key factor for all stakeholders, given the ultra-orphan nature of the patient population. Zevra owes modest milestones and mid-single-digit royalties to XOMA Corporation.