orphan drugs companies

Companies whose enriched business profile is tagged “orphan drugs”, drawn from their SEC filings + website text. Sorted by market capitalization; every figure is delayed data from SEC EDGAR + market data, shown with its as-of date.

• Insmed (INSM) Health Care — Insmed is a biopharmaceutical company dedicated to transforming the lives of patients suffering from serious and rare respiratory and inflammatory diseases. ask →
• Jazz Pharmaceuticals (JAZZ) Health Care — Jazz Pharmaceuticals is a biopharmaceutical company dedicated to developing innovative medicines for patients with complex sleep disorders and challenging oncology conditions. ask →
• TRAVERE THERAPEUTICS INC (TVTX) Health Care — A biopharmaceutical company developing and commercializing innovative therapies to address rare kidney and metabolic diseases with high unmet need. ask →
• ETON PHARMACEUTCIALS INC (ETON) Health Care — Eton Pharmaceuticals is a specialty drug company dedicated to acquiring and commercializing a diverse portfolio of therapies for rare diseases. ask →
• Ultragenyx (RARE) Health Care — Ultragenyx is a biopharmaceutical company dedicated to transforming the lives of patients suffering from rare and ultra-rare genetic diseases through innovative therapeutic development. ask →
• ZEVRA THERAPEUTICS INC (ZVRA) Health Care — Zevra Therapeutics is a rare-disease focused biotech company commercializing therapies for NPC and urea cycle disorders while advancing a late-stage pipeline. ask →
• CLIMB BIO INC (CLYM) Health Care — Climb Bio is a clinical-stage biotech developing anti-CD19 monoclonal antibodies to treat immune-mediated diseases like lupus and nephropathy. ask →
• AQUESTIVE THERAPEUTICS INC (AQST) Health Care — Aquestive Therapeutics develops advanced, non-invasive pharmaceutical delivery technologies and products, with a primary focus on its oral epinephrine prodrug candidates for severe allergic reactions. ask →
• FENNEC PHARMACEUTICALS INC (FENC) Health Care — Fennec Pharmaceuticals is a commercial-stage biopharmaceutical company providing the first and only FDA-approved therapy to prevent permanent hearing loss caused by cisplatin chemotherapy in pediatric cancer patients. ask →
• ROCKET PHARMACEUTICALS INC (RCKT) Health Care — Rocket Pharmaceuticals is a commercial-stage biotech company developing AAV-based gene therapies for rare cardiovascular conditions and genetic disorders. ask →
• ABEONA THERAPEUTICS INC (ABEO) Health Care — Abeona Therapeutics is a commercial-stage biopharmaceutical firm providing the first autologous cell-based gene therapy for the treatment of rare, debilitating RDEB skin wounds. ask →
• INHIBIKASE THERAPEUTICS INC (IKT) Health Care — A clinical-stage company developing a novel, oral anti-proliferative prodrug (IKT-001) for the treatment of Pulmonary Arterial Hypertension. ask →
• PROTALIX BIOTHERAPEUTICS INC (PLX) Health Care — Protalix BioTherapeutics leverages its proprietary plant cell-based protein expression platform to develop and commercialize life-saving enzyme replacement therapies for rare diseases. ask →
• SPRUCE BIOSCIENCES INC (SPRB) Health Care — A clinical-stage biopharmaceutical company advancing a breakthrough enzyme replacement therapy for the rare, neurodegenerative condition Sanfilippo Syndrome Type B. ask →
• KARYOPHARM THERAPEUTICS INC (KPTI) Health Care — Karyopharm Therapeutics develops novel cancer therapies using its proprietary XPO1 inhibition platform, with the lead drug XPOVIO currently used in multiple myeloma and a late-stage pipeline targeting myelofibrosis. ask →

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Data: SEC EDGAR (filings) + market data (fundamentals, delayed daily-close). Figures shown with their as-of date.

Last updated 2026-10-08.

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