Zevra Therapeutics

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

Zevra Therapeutics presented its plan to expand its presence in rare diseases at the Citizens Life Sciences Conference. In the fourth quarter, revenue rose 30% to $106.5 million, driven mainly by the commercial success of MIPLYFFA, which achieved a 40–50% market share in its first year. The company plans geographic expansion in Europe, aims to substantially increase the number of diagnosed NPC patients, and is recruiting patients for the celiprolol program targeting vascular Ehlers–Danlos syndrome. Zevra emphasizes disciplined capital allocation and the goal of financing sustainable growth independently of the capital markets. » More on de.investing.com