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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]
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".
According to Thomson Reuters in their 2012 publication "The Economic Power of Orphan Drugs", there has been increased investing in orphan drug research and development partly since the U.S. Congress enacted the Orphan Drug Act, giving an extra monopoly for drugs for "orphan diseases" that affected fewer than 200,000 people in the country. [13]
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 ...
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.
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 ...
BioMarin Pharmaceuticals (NAS: BMRN) keeps chugging along, pushing its orphan drug pipeline forward. Yesterday, the biotech announced data from two trials, giving the company confidence to move ...
Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical need. Fast track designation must be requested by the drug company.