Zevra Therapeutics is a commercial-stage rare disease pharmaceutical company. Its core product is MIPLYFFA (arimoclomol), which received FDA approval in September 2024 as the first treatment for Niemann-Pick disease type C (NPC), an ultra-rare progressive neurodegenerative disease affecting an estimated 900 patients in the U.S., of whom only 300-350 have been diagnosed. Zevra sells MIPLYFFA through specialty pharmacy channels, supported by a dedicated rare disease sales force calling on neurologists, geneticists, and pediatricians at NPC centers of excellence. Because the patient pool is tiny, revenue is driven by patient enrollment, conversion speed, and retention rather than volume — and pricing is calibrated accordingly at a high per-patient cost. Zevra also receives reimbursements from France for MIPLYFFA provided under expanded access, passive royalties from AZSTARYS (an ADHD drug fully licensed to Corium), and minimal revenue from OLPRUVA, a urea cycle disorder treatment where Zevra has pulled back investment and is evaluating strategic alternatives. In April 2025, Zevra sold a rare pediatric disease Priority Review Voucher received with the MIPLYFFA approval for net proceeds of $148M. Key pipeline assets include celiprolol, in a Phase 3 trial for vascular Ehlers-Danlos Syndrome, a rare connective tissue disorder with no approved treatments, and KP1077 for idiopathic hypersomnia, for which Zevra is evaluating strategic alternatives. Growth priorities include expanding the diagnosed U.S. NPC population through disease awareness and AI-driven patient identification, and pursuing EU approval for MIPLYFFA, where roughly 1,100 NPC patients are already largely diagnosed.