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 + Finnhub, shown with its as-of date.
- Alnylam Pharmaceuticals (ALNY) Health Care — A commercial-stage RNAi therapeutics pioneer with six FDA-approved siRNA medicines targeting rare genetic and cardiometabolic diseases. ask →
- Insmed (INSM) Health Care — A biopharmaceutical company with approved rare respiratory therapies (ARIKAYCE for MAC lung disease, BRINSUPRI for bronchiectasis) and a pipeline in respiratory, immunology, and neurological rare diseases. ask →
- MIRUM PHARMACEUTICALS INC (MIRM) Health Care — Mirum is a rare disease biopharmaceutical company commercializing approved bile acid and hepatic therapies while advancing late-stage candidates in cholestatic liver disease and viral hepatitis. ask →
- TRAVERE THERAPEUTICS INC (TVTX) Health Care — Rare kidney disease specialist with two approved FILSPARI formulations (IgAN, FSGS) and a clinical-stage enzyme therapy poised to become the first disease-modifying treatment for homocystinuria. ask →
- Ultragenyx (RARE) Health Care — A biopharmaceutical company developing and commercializing novel therapies for rare and ultra-rare genetic diseases with high unmet medical need. ask →
- SAVARA INC (SVRA) Health Care — Clinical-stage biopharmaceutical company developing treatments for rare and orphan respiratory diseases. ask →
- SOLID BIOSCIENCES INC (SLDB) Health Care — A gene therapy company advancing a pipeline of treatments for rare neuromuscular and cardiac diseases including Duchenne muscular dystrophy, Friedreich's ataxia, and cardiomyopathy. ask →
- AQUESTIVE THERAPEUTICS INC (AQST) Health Care — Aquestive develops orally disintegrating film formulations for CNS disorders, acute seizures, and opioid use, leveraging proprietary sublingual and buccal film platforms. ask →
- SPRUCE BIOSCIENCES INC (SPRB) Health Care — Clinical-stage biopharmaceutical company developing tralesinidase alfa enzyme replacement therapy for rare lysosomal storage disorders, particularly Sanfilippo Syndrome Type B. ask →
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Data: SEC EDGAR (filings) + Finnhub (fundamentals, delayed daily-close). Figures shown with their as-of date.
Last updated 2026-07-10.
Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & Finnhub, shown with their as-of date.