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Orphan Designation

The Orphan Drug Act (ODA) was signed into effect in the United States in 1983 to encourage the development of products treating diseases that affect fewer  than 200,000 people by offering certain benefits from the federal government. So far 9 million patients in America have benefited from products that stem from the ODA. Within the first 10 years after the ODA was signed 87 orphan drugs reached market, this was a huge increase compared to the 10 drugs created from 1974-1984.


Orphan Drug Act Benefits to Industry in the US

  • Orphan drugs receive fast-track for the FDA to evaluate registration files
  • FDA offers assistance to research protocol design
  • Phase III clinical trials can be conducted with less than 1000 patients enrolled
  • Companies receive 7-year marketing exclusivity on marketing authorization


Orphan Designations Around the World

Orphan conditions are a legal designation that allow different protections depending on the country. The two other area that designate Orphan Conditions are Europe and Japan.

The European equivalent to the Orphan Designation Act was passed in 2000 to offer benefits to a drug or a biologic product intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that does not affect more than 5 in 10,000 people. The European Union will assist with development of the medicine, reduce fees for marketing-authorization applications and make companies eligible for 10 year of marketing exclusivity.

Japan is the only country to also designate devices as Orphan. Japan will give Orphan Designation to vaccines, devices and drugs that treat incurable diseases affecting under 50,000 and that currently do not have a possible alternative treatment or if the proposed drug is proven to be clinically superior to the current treatment. Benefits issued in Japan include reduction in the required data, tax benefits, accelerated review, and extension of re-examination period.


AGTC Programs that have received Orphan Designation in the US and Europe

  • X-Linked Retinoschisis
  • X-Linked Retinitis Pigmentosa
  • Achromatopsia B3 and A3


Resources on Orphan Designation

Developing Products for Rare Diseases & Conditions

Designating an Orphan Product: Drugs and Biological Products

2017 Orphan Drug Report

Visionary science for life changing cures.

AGTC is now pre-screening for a phase 2/3 clinical trial of an investigational gene therapy in people with X-Linked Retinitis Pigmentosa.

Learn more about the clinical trial or how to qualify for pre-screening: