Definition of orphan drug in US English:

orphan drug


  • A pharmaceutical that remains commercially undeveloped owing to limited potential for profitability.

    • ‘When Congress and the FDA dreamed up the orphan drug bill they didn't consider limiting profitability.’
    • ‘One medical gas, nitric oxide, has been licensed as an orphan drug and increased in price.’
    • ‘These drugs are called orphan drugs and are allowed to have less premarketing testing.’
    • ‘The drug has been granted orphan drug status in American and Europe, providing an incentive for Amarin to complete its development.’
    • ‘In fact Glutathione is an orphan drug for the treatment of AIDS-associated cachexia.’
    • ‘It was given orphan drug status by the American Government because the expected uptake of the drug was quite small.’
    • ‘Upon successfully securing marketing approval from the FDA for this indication, Genetronics could then also secure orphan drug status, providing U.S. marketing exclusivity for seven years and certain tax benefits.’
    • ‘As an herbal product, the Hoxsey tonic cannot be patented and therefore occupies the status of an orphan drug that no company will develop.’
    • ‘This lesson has been learnt in the case of orphan drugs; we should generalise it to apply to other cases of market failure in research and development.’
    • ‘Orphan devices and combination products should be defined as products that address any disease or condition affecting less than 25,000 patients in the United States (for orphan drugs, the number is 200,000) per year.’
    • ‘The FDA approved 20 orphan drugs, biologics and devices in 1999 for the treatment of rare diseases with patient populations too small to make the development of such drugs and devices routinely profitable.’
    • ‘They didn't realize that they were developing the world's first billion-dollar orphan drug.’
    • ‘So leptin, therapeutically, is an orphan drug.’
    • ‘It's really an orphan drug which has benefited from the let's-give-it-a-shot mentality more than anyone thought possible.’
    • ‘Apokyn was designated an orphan drug in l991 to treat the ten percent, or about 112,000 Parkinson's patients who progress to stage four and experience the severe on/off motor fluctuations unresponsive to other therapies.’
    • ‘Intravenous L-carnitine is available as a prescription orphan drug for the treatment of primary and secondary L-carnitine deficiencies, and Propionyl-L-carnitne is available in Europe but not in the United States.’
    • ‘The orphan drug Vidaza which is given by injection for the treatment of Myelodysplastic Syndrome, a bone marrow disease, has been given approval by the U.S. Food and Drug Administration.’
    • ‘Arginox Pharmaceuticals announced that the US FDA has granted orphan drug designation to Tilarginine Acetate Injection for the treatment of cardiogenic shock.’
    • ‘It has has been designated an orphan drug in both the EU and the US, is currently under priority review in the US, Canada, Switzerland, Australia and New Zealand.’
    • ‘For identifying a potential pharmacologic treatment for cystinosis, demonstrating its effectiveness, and bringing it to market as an orphan drug, to completely prevent manifestations of this rare genetic inborn error of metabolism’


orphan drug

/ˈôrfən ˌdrəɡ/