Zevra Therapeutics

Zevra presented a rare-disease strategy at the Morgan Stanley Global Healthcare Conference and reported stronger early US uptake of MIPLYFFA. Over six quarters 184 prescription requests were recorded, corresponding to an estimated penetration of 52–61% of the diagnosed pool, and the company believes the addressable US market for NPC may be closer to 900 rather than 350 patients. Zevra ended Q2 with $260m in cash and says it is debt-free; the EMA re-examination of Arimoclomol is expected in Q4 2024. The phase III study of celiprolol for vascular Ehlers–Danlos is progressing more slowly with lower event rates than expected, so further discussions with the FDA are planned. » More on de.investing.com

Zevra Therapeutics unveiled an expanded growth plan at the Canaccord Growth Conference that centers on MIPLYFFA for Niemann‑Pick type C as the commercial focus and accelerated development of celiprolol for vascular Ehlers‑Danlos. In the US the company recorded 184 cumulative requests for enrollment in the prescription program; about 50% of patients are adults, so the addressable market is estimated at roughly 350–900 prevalent US patients. Zevra holds $260 million in cash, is debt‑free, and plans an EMA resubmission after a negative opinion while holding talks with the FDA to accelerate celiprolol. MIPLYFFA will be priced with a WAC of $85,000 per patient per month; management expects further growth from increased diagnostic activity and views competition as potentially market‑expanding. » More on de.investing.com

Zevra reported net revenue of $39.7 million and earnings of $0.14 per share for the second quarter of 2026, with both metrics above analyst estimates. US sales of MIPLYFFA rose to $30.2 million (+40% year‑on‑year) and drove overall year‑on‑year growth of 53%. The company holds $260.2 million in cash and cash equivalents and is focused on a renewed review of the European marketing authorisation application after a negative CHMP opinion. Management cited further priorities as expanding diagnostics, reach among prescribing physicians and access support; no formal revenue guidance was provided. » More on de.investing.com