Are orphan drugs approved drugs?
Orphan drugs may be approved or experimental. The term “orphan drug” can refer to either a drug or biologic intended for use in a rare disease or condition. A drug or biologic becomes and “orphan drug” when it receives orphan-drug designation from the Office of Orphan Products Development at the FDA. Orphan-drug designation qualifies the sponsor to receive certain benefits from the Government in exchange for developing the drug for a rare disease or condition. The drug must then go through the FDA marketing approval process like any other drug or biologic which evaluates for safety and efficacy. To date over 1400 drugs and biologics have been designated as orphan-drugs, and over 250 have been approved for marketing.
