EU panel rejects Zevra's marketing application for arimoclomol
Shares of drugmaker Zevra Therapeutics ZVRA.O fell 31.2% to $8.64 in premarket trading.
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.
The company 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 said 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 the last close, the stock was up 39.7% year to date.
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