EU regulators back Minoryx’s ALD therapy while rejecting Zevra’s rare-disease drug

The EU Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on Minoryx Therapeutics' drug for adrenoleukodystrophy, marking a significant regulatory step after previous challenges.

In the same meeting, the CHMP delivered a negative opinion on Zevra's Meplyffa, a treatment for a rare disease, stopping its progress toward European approval despite earlier US clearance.

The contrasting decisions underscore the distinct regulatory pathways in Europe, giving Minoryx a path toward market entry while leaving Zevra to address the committee's concerns before any re‑submission.