rare disease therapeutics companies
Companies whose enriched business profile is tagged “rare disease therapeutics”, 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.
- Alnylam Pharmaceuticals (ALNY) Health Care — Alnylam is a pioneer in RNA interference (RNAi) therapeutics, using gene-silencing technology to treat rare genetic diseases and cardiovascular conditions. ask →
- Jazz Pharmaceuticals (JAZZ) Health Care — A rare-disease biopharmaceutical company with diversified marketed therapies in epilepsy, sleep disorders, and oncology, and an expanding pipeline of innovative treatments. ask →
- KINIKSA PHARMACEUTICALS INTERNATIO (KNSA) Health Care — Kiniksa is a biopharmaceutical company commercializing ARCALYST (IL-1 cytokine trap) for recurrent pericarditis and rare inflammatory diseases, with a pipeline of next-generation IL-1R1 inhibitors in clinical development. ask →
- CATALYST PHARMACEUTICALS INC (CPRX) Health Care — Catalyst Pharma commercializes rare disease therapies with a focus on neuromuscular and neuromuscular-inflammation disorders, generating strong cash flow from established and newly approved products. ask →
- PALVELLA THERAPEUTICS INC (PVLA) Health Care — A clinical-stage biopharmaceutical company developing proprietary therapeutics for rare skin diseases and vascular malformations following the merger of Pieris Pharmaceuticals and Legacy Palvella. ask →
- WAVE LIFE SCIENCES LTD (WVE) Health Care — A clinical-stage biotechnology company developing a versatile, multimodal RNA medicine platform to treat rare and common diseases without the need for complex delivery vehicles like lipid nanoparticles. ask →
- LARIMAR THERAPEUTICS INC (LRMR) Health Care — Larimar Therapeutics is developing a novel cell-penetrating peptide platform to treat rare, genetically driven diseases, with a lead program targeting the underlying protein deficiency in Friedreich's ataxia. ask →
- FENNEC PHARMACEUTICALS INC (FENC) Health Care — Commercial-stage biopharma company bringing PEDMARK, the first and only approved drug to prevent cisplatin-induced ototoxicity in pediatric cancer patients, to market. ask →
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Frequently asked
What are the largest rare disease therapeutics companies?
By market capitalization, the largest rare disease therapeutics companies in the catalog include Alnylam Pharmaceuticals (ALNY), Jazz Pharmaceuticals (JAZZ), KINIKSA PHARMACEUTICALS INTERNATIO (KNSA), CATALYST PHARMACEUTICALS INC (CPRX), PALVELLA THERAPEUTICS INC (PVLA).
How many rare disease therapeutics companies does stocks-llm track?
stocks-llm currently lists 8 rare disease therapeutics companies (as of 2026-07-31), each with dated fundamentals from SEC EDGAR and market data. Informational only — not financial advice.
Data: SEC EDGAR (filings) + market data (fundamentals, delayed daily-close). Figures shown with their as-of date.
Last updated 2026-07-31.
Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & market data, shown with their as-of date.