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 →
• 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 →
• XERIS BIOPHARMA HOLDINGS INC (XERS) Health Care — A commercial-stage biopharma company with three FDA-approved therapies for rare endocrine and neurological diseases and a proprietary injectable drug formulation platform for rare diseases. ask →
• OMEROS CORP (OMER) Health Care — A clinical-stage biotech company commercializing complement-pathway inhibitors for rare hematologic and immune diseases, with FDA-approved YARTEMLA for stem cell transplant complications and a $240M asset sale of its lead MASP-3 program to Novo Nordisk. ask →
• SAVARA INC (SVRA) Health Care — Savara is a clinical-stage biopharmaceutical company dedicated to developing innovative inhalable therapies for rare 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 →
• GALECTIN THERAPEUTICS INC (GALT) Health Care — Clinical-stage biopharmaceutical company developing galectin-3 inhibitors derived from plant carbohydrates to treat liver fibrosis, cirrhosis, and cancer. ask →
• AN2 THERAPEUTICS INC (ANTX) Health Care — AN2 Therapeutics leverages a proprietary boron chemistry platform to develop small-molecule drugs for neglected infectious diseases, blood cancers, and other high-unmet-need conditions. 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) + market data (fundamentals, delayed daily-close). Figures shown with their as-of date.

Last updated 2026-08-24.

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