orphan drug
A medicinal drug which is effective in the treatment of some disease(s), but which is not manufactured or marketed because the demand is insufficient to cover the costs of supply.
Definitions
A medicinal drug which is effective in the treatment of some disease(s), but which is not manufactured or marketed because the demand is insufficient to cover the costs of supply.
Tragically, her disease is so rare that the only effective medicine is an orphan drug.
Comments
(US, legal) A drug as defined under the Orphan Drug Act (1983).
Tragically, her disease is so rare that the only effective medicine is an orphan drug.
“European regulators back new DMD gene therapy with key status https://www.europesays.com/europe/32461/ Regulators in the European Union have granted orphan drug designation to SGT-003, a gene therapy for Duchenne muscular…”
— Bluesky· May 2026
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