Zevra tanks after EU panel's negative opinion on rare disease drug

Zevra Therapeutics

24/07/2026 13:18

** Shares of drugmaker Zevra Therapeutics ZVRA.O fall 31.2% to $8.64 premarket
** The European Medicines Agency's CHMP adopted a negative opinion on Zevra Therapeutics' marketing application for arimoclomol, saying the treatment's efficacy had not been sufficiently demonstrated
** Co was seeking approval to treat Niemann-Pick disease type C, a rare genetic disorder that causes fatty substances to build up in cells, leading to progressive damage to the brain and other organs
** Zevra says it plans to request a re-examination of the CHMP's decision under the EU regulatory process and will continue providing access to arimoclomol for eligible patients through its global Expanded Access Program
** Zevra Denmark can request a re-examination of the CHMP's negative opinion within 15 days of receiving the decision
** The U.S. FDA approved arimoclomol, branded as Miplyffa, in 2024 as the first treatment for the condition, carrying an annual price of just over $1 million at the highest dose
** As of last close, stock up 39.7% YTD

 (Reporting by Kamal Choudhury in Bengaluru)
 ((Kamal.Choudhury@thomsonreuters.com;))