ZVRA - Negative CHMP Opinion; Company to Pursue Re-Examination With Confidence in Remaining Issues

Research evidence excerpt

ZVRA - Negative CHMP Opinion; Company to Pursue Re-Examination With Confidence in Remaining Issues

July 24, 2026

Eddie Hickman, Ph.D. eddie.hickman@guggenheimpartners.com ZVRA - Negative CHMP Opinion; Company to Pursue

212 518 9904 Re-Examination With Confidence in Remaining

Issues

ZVRA BUY Key Message: We caught up with Zevra management following this morning's

Zevra Therapeutics, Inc. announcement of a negative CHMP opinion on the arimoclomol MAA, and the company

Sector: Biotechnology intends to formally request re-examination. Per the CHMP's public statement, the

negative opinion was driven by (1) uncertainties about the reliability and robustness of

Price Target Change results due to data handling and analytical choices, (2) lack of benefit in ambulation and

Share Price $12.52 cognition endpoints within the 5-domain NPCCSS, and (3) a positive treatment effect in

Price Target $24.00 the miglustat-combination subgroup that was not considered robust given the absence

Prior $29.00 of effect in the overall population. Management indicated that all previously raised issues

have been overcome through the review process, with the only remaining issues on the

CHMP's interpretation of the pivotal study analysis, which they believe is addressable

Revenue ($M) through additional analyses under the CHMP's own framework, with no new clinical data

(FY DEC) 1Q 2Q 3Q 4Q FY required. The re-examination process runs ~120 days, with a new set of rapporteurs

2026 36.2 40.3E 42.3E 46.3E 164.0E assigned (4Q26 update). Management is not saying yet which specific avenues they

2027 — — — — 185.0E will pursue, but points to the recent Acadia Daybue reversal as a clear precedent

Prior — — — — 207.5E that a well-constructed re-examination can overcome the remaining hurdles. While the