ORPHAN DRUG and ORPHAN DISEASE
An orphan drug is a pharmaceutical agent that has been developed specially to treat a rare medical condition; the condition itself being referred to as an orphan disease. Orphan disease is a disease that affects a small percentage of the population.
Examples of orphan disease & drugs:
Wilson’s disease: Penicillamine was developed to treat wilson’s disease, a rare hereditary disease that can lead to a fatal accumulation of copper in the body. The drug was later found to be effective in treating arthritis.
Cystic fibrosis: Cystic fibrosis is an autosomal recessive genetic disorder affecting most critically the lungs & also the pancreas, liver & intestine. In the 1980s, cystic fibrosis patients are rarely found. Drugs like pulmozyme & tobramycin both developed that significantly improve their quality of life.