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ZEVRA THERAPEUTICS INC (ZVRA)

Health Care · NASDAQ

Zevra brings life-changing therapies to ultra-rare neurodegenerative and metabolic diseases, with FDA-approved MIPLYFFA for Niemann-Pick disease type C now generating revenue and a pipeline of clinical candidates addressing severe unmet needs.

What ZEVRA THERAPEUTICS INC does

Zevra Therapeutics is a commercial-stage biopharmaceutical company focused on rare diseases with limited or no treatment options. The company markets MIPLYFFA (arimoclomol) for Niemann-Pick disease type C and OLPRUVA (sodium phenylbutyrate) for urea cycle disorders, while advancing celiprolol in Phase 3 trials for Vascular Ehlers-Danlos syndrome and developing serdexmethylphenidate-based candidates for idiopathic hypersomnia and narcolepsy. The company also receives royalties and milestones from its partnered ADHD therapy AZSTARYS.

Themes: ["Rare disease / orphan drugs","Niemann-Pick disease type C","Urea cycle disorders","Vascular Ehlers-Danlos syndrome","Idiopathic hypersomnia","Narcolepsy","ADHD (royalty partnership)","Neurodegenerative diseases","Metabolic disorders"]

Fundamentals

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Key risks (from latest filing)

["Dependence on commercial success of MIPLYFFA, which has limited patient population (approximately 300-350 diagnosed in US), and global expansion competition with existing NPC treatments in mature European markets","Clinical development risks for celiprolol Phase 3 trial and serdexmethylphenidate candidates, with potential for trial delays, failure to meet efficacy endpoints, or regulatory rejection","Reliance on royalties and milestones from partnered AZSTARYS product, with no direct revenue control and dependence on partner's commercialization efforts"]

See ZVRA's biggest risks from its latest 10-Q →

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Fundamentals: Finnhub, as of 2026-07-10. Filings: SEC EDGAR. Prices are delayed daily-close data.

Last updated 2026-07-09.

Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & Finnhub, shown with their as-of date.