
Zevra Therapeutics added a Miplyffa patent to the FDA Orange Book, extending exclusivity to 2039. The NP-C drug, approved last September, now covers roughly 900 potential U.S. patients.
Shares of Zevra Therapeutics jumped 29% over two sessions after the company added a patent for its Niemann-Pick Disease Type C therapy Miplyffa to the FDA Orange Book. The drug's patent protection now extends to 2039, a filing showed.
The Orange Book listing strengthens Zevra's exclusivity position on Miplyffa, which won FDA approval in September 2024 as the first oral treatment for the rare genetic disorder. NP-C affects roughly 900 patients in the U.S., and the drug carries an annual list price near $500,000 before patient assistance programs.
Zevra ended the second quarter with $76.4 million in cash and equivalents, enough to fund operations into 2026, the company said in its August earnings release. The stock still trades about 40% below its 52-week high set in January, when the company's initial Miplyffa launch covered fewer than 20 patients.
The patent move follows Zevra's July acquisition of the drug's original developer, Aadi Bioscience, for roughly $71 million in stock. CEO Neil F. McFarlane told analysts on the Q2 call that the company expects to treat 30 to 40 patients by year-end, with a full sales force now deployed.
A counter-party risk remains, the company said: a competing NP-C therapy in Phase 2 trials could narrow Miplyffa's addressable market if it reaches approval. Zevra shares closed at $5.82 on Wednesday.
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