Orphan Drug Designation

The United States Orphan Drug Act created the orphan drug designation program to provide financial incentives to sponsors developing drugs and biologics for rare diseases and conditions, which are generally defined as affecting fewer than 200,000 persons in the United States.  Orphan status is granted by the Office of Orphan Products Development (OOPD) at the Food and Drug Administration (FDA) under the Federal Drug and Cosmetic (FD&C) Act, Section 526, Part B 360bb and Part C 360cc. 

The benefits of orphan drug designation include various incentives during development, and potential marketing exclusivity after FDA approval. The benefits of orphan drug status include:

  • 50% tax credit on the cost of qualified clinical trials conducted within the US
  • Seven-year marketing exclusivity for the active ingredient following FDA marketing approval
  • Priority review of a New Drug Application 
  • Waiving of review fees for the New Drug Application
  • Waiving of PREA (Pediatric Research Equity Act) study requirements

In Europe, the European Medicines Agency allows all orphan medicines to be assessed for marketing authorisation centrally, permitting companies to have a single decision valid across all European Union member countries. An authorised orphan medicine also provides a potential benefit of 10 years of marketing exclusivity once approved, preventing similar, competing drugs from being approved for the same therapeutic indication.

Orphan designation is also available in Japan under similar criteria and conditions.

Multiple System Atrophy and Progressive Supranuclear Palsy are considered orphan diseases and have the potential for orphan designation.