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According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US" (which equates to approximately 6 cases per 10,000 population) "or meets cost recovery provisions of the act".
In contrast, the decade prior to 1983 saw fewer than ten such products come to market. From the passage of the ODA in 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds. As of 2010, 200 of the roughly 7,000 officially designated orphan diseases have become treatable. [13]
Lists of all drugs that have received orphan status in the United States and Europe are available from the U.S. Food and Drug Administration and the European Commission, respectively: FDA List of Orphan Designations and Approvals [dead link ] European Commission Register of designated Orphan Medicinal Products
TransCon PTH (palopegteriparatide) originally received Orphan Drug Designation from the U.S. FDA in June 2018. The FDA grants orphan designation to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States, and that potentially ...
The FDA has granted Orphan Drug Designation to Paratek Pharmaceuticals Inc's (NASDAQ: PRTK), Nuzyra (omadacycline) for infections caused by Nontuberculous Mycobacteria (NTM). The orphan drug ...
Since being signed into law 30 years ago, the Orphan Drug Act, or ODA, has resulted in the approval of more than 400 new drugs and biologic products in the United States. The goal of the ODA is to ...
Add yet another to the wave of orphan drugs preparing to hit the market. Pfizer (NYS: PFE) announced that the FDA has approved bosutinib as an orphan drug for chronic myelogenous leukemia, or CML.
In 2019, a drug named ARCT-810 which uses Lunar, received FDA orphan drug status for treating ornithine transcarbamylase deficiency. [17] The orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States, and grants seven years of marketing exclusivity after approval. [18]