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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
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]
Cardiac valvular disease, pulmonary hypertension, cardiac fibrosis; [3] [23] re-approved in June 2020 for the treatment of seizures associated with Dravet syndrome, under FDA orphan drug rules. Fenoterol: 1990 New Zealand Asthma mortality. [3] Feprazone: 1984 Germany, UK Cutaneous reaction, multiorgan toxicity. [3] Fipexide: 1991 France ...
Lucinactant is listed as an Orphan Drug Product by the US Food and Drug Administration for several conditions: [9] [10] 07-30-1996 Treatment of meconium aspiration syndrome in newborn infants; 07-17-1995 Treatment of acute respiratory distress syndrome in adults. 05-23-2006 Prevention of bronchopulmonary dysplasia in premature infants
Orphan drug status, which is given to drugs meant for the treatment of rare diseases, offers drug developers a path to faster approval and enhanced market exclusivity. So let's look at a small ...
The FDA grants orphan drug and rare pediatric disease designations to Ionis' (IONS) ION582 for treating Angelman syndrome. These designations will help incentivize development of the candidate.
Gilteritinib was granted orphan drug status by the U.S. FDA, the European Commission (EC) and the Japan Ministry of Health, Labor and Welfare, for some AML patients. [12] Gilteritinib was approved for medical use in Australia in March 2020. [13]