Zevra Therapeutics shares fell 25.4 percent after the EMA's CHMP rejected arimoclomol for Niemann-Pick disease type C in a re-examination bid. The negative opinion triggered a securities investigation and cast uncertainty over the company's European expansion plans.
Zevra Therapeutics shares fell 25.4 percent after the European Medicines Agency's CHMP rejected arimoclomol for Niemann-Pick disease type C in a re-examination bid.
"Longitudinal real-world studies can provide valuable insights that complement findings from clinical trials, particularly in ultra-rare diseases such as NPC," Elizabeth Berry-Kravis, MD, lead author of a new Zevra study and professor at Rush University Medical Center, said.
The negative opinion from the Committee for Medicinal Products for Human Use, issued July 24, triggered a securities investigation by Levi & Korsinsky on behalf of investors who relied on public statements about arimoclomol's approval status. The stock has since fallen approximately 25 percent from pre-announcement levels.
The setback follows the U.S. Food and Drug Administration's approval of MIPLYFFA (arimoclomol) on Sept. 20, 2024, for NPC in combination with miglustat. In the Phase 3 trial, MIPLYFFA halted disease progression compared with placebo over the one-year study period when measured by the NPC Clinical Severity Scale, the only validated disease progression tool for the condition.
Zevra published four-year real-world data from its U.S. Early Access Program in Molecular Genetics and Metabolism on Aug. 3, covering 109 participants across 14 sites, including 53 adults (48.6 percent) — the first published real-world evaluation of arimoclomol in adults with NPC. Mean arimoclomol exposure was 820 days, with 71 participants (65.1 percent) receiving concomitant miglustat. Disease severity scores remained relatively stable across follow-up through years one through four, and safety observations were consistent with the established profile. Common adverse reactions in the Phase 3 trial included upper respiratory tract infection, diarrhea, and decreased weight, each occurring in at least 15 percent of treated patients.
The company has accumulated more than five years of patient experience across 270-plus NPC patients worldwide through its Phase 2/3 trial, open-label extension, expanded access programs, and a pediatric sub-study — the most expansive clinical development program in NPC to date.
The planned re-examination keeps the EU opportunity alive but with reduced probability and unclear timing. Community fair value estimates for ZVRA range from approximately $12 to over $80 per share, reflecting wide divergence on the European revenue potential. The company's recent profitability and low earnings multiple remain core elements of the bull case.
The CHMP re-examination outcome will determine whether Zevra can access the European NPC market, where the drug holds Orphan Medicinal Product designation. Investors will watch for the re-examination timeline and any updates on the securities investigation in coming weeks.
This article is for informational purposes only and does not constitute investment advice.
