FDA Designates Orphan Drug Status for Rare Diseases
The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. At the time of the enactment of the Orphan Drug Act, the United States Congress made several factual findings. For example, the Congress observed that there are many diseases and conditions, such as Huntington’s disease, ALS (Lou Gehrig’s disease), Tourette syndrome, and muscular dystrophy, for example, which affect such small numbers of individuals that the diseases and conditions are considered rare in the United States. As a result, adequate drugs have not been developed and realistically cannot be expected to be developed because companies would likely incur a financial loss in the pursuit of such drugs, given the high cost of developing drugs.
Back in the early 1980s when the Orphan Drug Act passed, Congress made the determination that a seven (7) year period of exclusivity was appropriate if a company produced a drug to treat a rare disease that affects less than 200,000 people in the United States. Orphan designation is also possible if a disease affects more than 200,000 people if it can also be shown that there is no reasonable expectation that the cost of developing the drug could be recouped absent this period of exclusivity. This “patent-like” exclusivity is available even if the drug is not novel and non-obvious.
Orphan Drug Designation for Total Body Irradiation Treatment
Soligenix, Inc. (OTCQB: SNGX), a development stage biopharmaceutical company, recently announced that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted orphan drug designation to OrbeShield™ (oral beclomethasone 17,21-dipropionate or oral BDP), which is for the prevention of death following a potentially lethal dose of total body irradiation during or after a radiation disaster.
Oral BDP is a highly potent, topically active corticosteroid that has a local effect on inflamed tissue. BDP is the active ingredient in OrbeShield™, currently being developed for the treatment of gastrointestinal acute radiation syndrome (GI ARS), the driving component in early mortality following high doses of total body irradiation (TBI).
01.7.13 | posts | Gene Quinn
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10.28.13 | Patent Issues | Gene Quinn