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• Zevra plans to request a re-examination and will submit a detailed response.
• H.C. Wainwright called the negative opinion on Zevra Therapeutics’ marketing application for arimoclomol “a disappointing outcome that clearly increases the regulatory risk around the European opportunity.”
• Arimoclomol is already approved and marketed in the U.S. under the brand name MIPLYFFA.

Shares of Zevra Therapeutics (ZVRA) fell by as much as 27% on Friday but pared some losses after H.C. Wainwright noted that the company’s recent setback for its drug arimoclomol in Europe was “not insurmountable.”

At the time of writing, the stock was down 23%, on track for its worst day since March 2021.

ZVRA’s Setback

On Friday, Zevra announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion on its application to market arimoclomol for Niemann-Pick disease type C (NPC), a rare progressive neurodegenerative disorder.

The committee concluded that the drug’s effectiveness had not been clearly proven. It cited uncertainties in how the trial data were handled and analyzed, and noted no clear benefit in walking ability or cognition. Although patients already taking miglustat, alongside arimoclomol, showed some positive signs, these were not considered robust enough for the overall population. As a result, the benefits did not outweigh the risks, the committee said.

Zevra plans to request a re-examination and will submit a detailed response. CEO Neil F. McFarlane said the company remains “confident in the totality of evidence” and is committed to finding pathways to bring the treatment to European patients.

Analyst Weighs In

H.C. Wainwright called the negative opinion on Zevra Therapeutics’ marketing application for arimoclomol “a disappointing outcome that clearly increases the regulatory risk around the European opportunity.” The firm maintained its ‘Buy’ rating and $26 price target, noting that Zevra plans to request a re-examination and that this is not the end of the process. Analysts left estimates and valuation unchanged ahead of the company’s August 5 earnings call.

Arimoclomol is already approved and marketed in the U.S. under the brand name MIPLYFFA. The FDA cleared it in September 2024 as the first treatment for neurological symptoms of NPC in patients aged 2 years and older, used in combination with miglustat. MIPLYFFA generated $87.4 million in net revenue for the full year 2025 and accounted for a lion’s share of the company's total net revenue of $106.5 million.

How Did ZVRA Retail Traders React?

On Stocktwits, retail sentiment around ZVRA stock stayed within the ‘extremely bullish’ territory over the past 24 hours, while message volume rose from ‘high’ to ‘extremely high’ levels.

A Stocktwits user noted that Zevra has not provided details pertaining to the timeline for its further submission.

Another opined that Zevra’s success may come down to whether its drug is superior to privately held pharma company IntraBio’s Aquneursa, also approved for Niemann-Pick disease Type C.

ZVRA stock has gained 8% year-to-date. 

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