The Food and Drug Administration's orphan drug designation aims to accelerate the development and approval of crucial medicines to treat rare diseases that affect fewer than 200,000 Americans.
Drug companies who earn the designation for a rare disease treatment receive a seven-year drug exclusivity period, tax credits for up to 50 percent of research and development costs and waived regulatory fees, among other incentives.
Here are six drugmakers who've earned an orphan drug designation in 2017.
- Propanc Biopharma received the designation for PRP, a once-daily intravenous drug to treat pancreatic cancer.
- The FDA granted orphan status to Abeona Therapeutics' ABO-201 program, a gene therapy program for juvenile Batten disease.
- Mallinckrodt earned the designation for its experimental Duchenne muscular dystrophy treatment MNK-1411.
- Conatus Pharmaceuticals received orphan drug status for IDN-7314, a treatment for primary sclerosing cholangitis, which afflicts the body's bile ducts and can cause cirrhosis and liver failure.
- The FDA granted Epizyme's soft tissue sarcoma drug tazemetos orphan drug designation.
- Zogenix earned orphan drug status for ZX008, which is used to treat a rare form of epilepsy called Lennox Gastaut Syndrome.
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