Definition: An orphan drug is one that has been developed to treat a rare disease, defined by the FDA as a disease that affects fewer than 200,000 Americans.
In 1983, the Orphan Drug Act was passed to stimulate research, development and approval of drugs for those Americans who suffered from rare diseases. The act made the development of rare diseases drugs more financially beneficial to the companies that would develop them. Since then, more than 100 orphan drugs have been developed.
Examples:
Among the orphan drugs developed and marketed in the United States are drugs such as:
- Humira/Adalimumab used for the treatment of rheumatoid arthritis.
- Abelcet/Amphotericin B lipid complex used for invasive fungal infections.
- Botox/Botulinum toxin type A approved for treatment of cervical dystonia, used off-label for reducing facial wrinkles and other purposes.
