Rare and Orphan Disease Companies

U.S. Exchanges

List of Publicly Traded Companies Focusing on Rare Diseases Listed on U.S. Exchanges

The publicly traded companies on this list are biotech, medical device, pharmaceutical or other healthcare companies that provide products for rare diseases (also known as orphans disease). While the number varies worldwide, in the United States a rare disease is typically defined as a disease that affects fewer than 200,000 people.


Additional publicly traded healthcare companies and healthcare categories can be accessed through the link below:

List of Publicly Traded Healthcare Companies

A comparison widget that shows trend, earnings per share (EPS), P/E ratio and beta for each of the companies on this list can be accessed through the link below.

Rare and Orphan Disease Industry Comparison Widget

Select the company’s link to access charts, news links and company website and social media information.

Rare Diseases: Large-Cap Stocks

Alexion Pharmaceuticals, Inc. (ALXN) (Biopharmaceutical company: nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, hypophosphatasia, Lysosomal Acid Lipase Deficiency)

BioMarin Pharmaceutical Inc. (BMRN) (Therapies for serious and life-threatening rare and ultra-rare genetic diseases)

Rare Diseases: Mid-Cap Stocks

Horizon Pharma plc (HZNP) (Ireland, biopharmaceutical company: arthritis pain, rare diseases)

Intercept Pharmaceuticals, Inc. (ICPT) (Biopharmaceutical company: orphan and more prevalent liver diseases)

Ionis Pharmaceuticals, Inc. (IONS) (RNA-targeted drug discovery and development: cancer, cardiovascular, metabolic, neurological disorders, rare diseases)

Sarepta Therapeutics, Inc. (SRPT) (Biopharmaceutical company: RNA-based therapeutics; rare and infectious diseases)

Ultragenyx Pharmaceutical Inc. (RARE) (Biopharmaceutical company: debilitating genetic diseases)

Rare Diseases: Small-Cap Stocks

Acceleron Pharma Inc. (XLRN) (Cancer and orphan diseases)

Agios Pharmaceuticals, Inc. (AGIO) (Biopharmaceutical company: cancer and rare genetic disorders of metabolism)

Amicus Therapeutics, Inc. (FOLD) (Biopharmaceutical company: lysosomal storage diseases)

Audentes Therapeutics, Inc. (BOLD) (IPO July 20, 2016: biotech company focused on therapy treatments for rare diseases)

Avexis Inc. (AVXS) (IPO February 11, 2016: Gene therapy company with a focus on rare neurological genetic diseases)

Bluebird Bio, Inc. (BLUE) (Gene therapies for severe genetic and rare diseases)

Insmed, Inc. (INSM) (Biopharmaceutical company: lung disease and pulmonary arterial hypertension)

Raptor Pharmaceutical Corp. (RPTP) (Biopharmaceutical company: nephropathic cystinosis)

Spark Therapeutics, Inc. (ONCE) (Gene therapies: treatments for debilitating genetic diseases)

Rare Diseases: Micro-Cap Stocks

Abeona Therapeutics (ABEO) (Gene therapy and plasma-based products)

Adverum Biotechnologies, Inc. (ADVM) (Biopharmaceutical company: serious eye diseases)

Aegerion Pharmaceuticals, Inc. (AEGR) (Biopharmaceutical company: homozygous familial hypercholesterolemia)

Akari Therapeutics, Plc (AKTX) (United Kingdom, biopharmaceutical company: therapeutics to treat orphan autoimmune and inflammatory diseases)

aTyr Pharma Inc. (LIFE) (IPO in MAY 2015: Medicines used in the treatment of severe rare diseases)

Catabasis Pharmaceuticals Inc. (CATB) (Biopharmaceutical company: rare diseases, serious lipid disorders)

Catalyst Pharmaceuticals, Inc. (CPRX) (Biopharmaceutical company: rare neuromuscular and neurological diseases)

Chiasma Inc. (CHMA) (IPO in July 2015, biopharmaceutical company: oral versions of injected drugs for orphan diseases)

Corbus Pharmaceuticals Holdings, Inc. (CRBP) (Biopharmaceutical company: rare, life-threatening, inflammatory diseases)

Dimension Therapeutics Inc. (DMTX) (IPO in October 2015: Gene therapy platform company: rare diseases associated with the liver)

Fate Therapeutics, Inc. (FATE) (Biopharmaceutical company: cellular immunotherapeutics; cancer, rare genetic diseases and immune disorders)

Idera Pharmaceuticals, Inc. (IDRA) (B-cell Lymphoma, autoimmune, rare diseases)

Medgenics, Inc. (MDGN) (Ex vivo gene therapy platform)

MyoKardia Inc. (MYOK) (IPO in October 2015: Biopharmaceutical company; therapies for serious and neglected rare cardiovascular diseases)

PTC Therapeutics, Inc. (PTCT) (Biopharmaceutical company: oral treatments)

Strongbridge Biopharma plc (SBBP) (IPO in October 2015: Biopharmaceutical company; rare endocrine disorders and other rare diseases)

Summit Therapeutics Plc. (SMMT) (IPO in March 2015: United Kingdom, biopharmaceutical company: Duchenne Muscular Dystrophy, infectious disease caused by the bacteria C. difficile)

UniQure N.V. (QURE) (Netherlands: gene therapy)

Xenon Pharmaceuticals Inc. (XENE) (Canada, biopharmaceutical company: lipoprotein lipase deficiency)

Rare Diseases: Nano-Cap Stocks

Bio Blast Pharma Ltd. (ORPN) (Israel: therapies for patients with rare and ultra-rare genetic diseases)

Eiger BioPharmaceuticals, Inc. (EIGR) (Biopharmaceutical company: Hepatitis Delta, Hypoglycemia, Pulmonary Arterial Hypertension, Lymphedema)

Phaserx, Inc. (PZRX) (IPO May, 18 2016: biopharmaceutical company focused on treatments for enzyme deficiencies in the liver)

Related Links:

List of Biotech Companies

Publicly Traded Companies by Sector and Industry

List of Pharmaceutical Companies