FDA Convenes Expert Panel Meeting For Zevra Therapeutics' Arimoclomol, Analyst Optimistic About Approval Despite Small Patient Base

The FDA will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2 to review the marketing application for Zevra Therapeutics Inc.’s ZVRA arimoclomol for Niemann-Pick disease type C (NPC).

The application for arimoclomol has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, which was extended from June 21.

Previously known as KemPharm, Zevra Therapeutics acquired arimoclomol from Orphazyme for $12.8 million. 

In 2021, the FDA issued a complete response letter to arimoclomol as a treatment option for Niemann-Pick disease type C.

The CRL asked for additional qualitative and quantitative evidence to further support the validity and interpretation of the 5-domain NPC Clinical Severity Scale, particularly the swallow domain.

William Blair says the data supports the efficacy and safety of arimoclomol supports approval and expects significant patient advocacy at the meeting in support of approval.

Additionally, unlike the prior filing for arimoclomol, patient groups have already sent letters of support, including a petition from the National Niemann-Pick Disease Foundation and six other NPC advocacy and research organizations with nearly 1,000 signatures from NPC patients, caregivers, and physicians.

If approved, arimoclomol would represent a significant commercial opportunity for Zevra despite the ultra-rare nature of NPC. The analyst adds that NPC is believed to affect around 900 patients in the U.S., of whom an estimated 300-400 are currently diagnosed.

The company established a commercial infrastructure for launching Olpruva earlier this year. With about 70 patients on therapy through the early access program in the U.S., the analyst expects a strong launch.

This will involve converting these patients to full-launch participants and attracting new patients, many of whom have already shown interest.

Zevra owes modest milestones and mid-single-digit royalties to XOMA Corporation XOMA. Given the ultra-orphan nature of the patient population, pricing will be a key variable for all stakeholders involved, pending approval.

William Blair reiterates the Outperform rating.

Price Action: ZVRA shares are up 17.4% at $5.27 at last check Tuesday.