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Thursday, April 24, 2014

FDA Grants Orphan Drug Status to Alvocidib, 2nd Action in AML this Month


The U.S. Food and Drug Administration has granted orphan drug designation to Alvocidib (flavopiridol) to treat patients with acute myeloid leukemia. Alvocidib, a potent cyclin-dependent kinase small molecule inhibitor, is being evaluated in patients with intermediate or high-risk AML, who typically have a poor prognosis due to limited treatment options.


The news comes after the FDA granted another AML drug, Volasertib, orphan drug designation earlier this month.


The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.


About 18,860 new cases of AML are expected to be diagnosed this year, according to estimates from the American Cancer Society. The average age at diagnosis is 66.


Alvocidib has been evaluated in multiple Phase II clinical trials involving approximately 400 patients with both relapsed/refractory or frontline, previously untreated intermediate and high-risk AML. The trials have tested the drug as a single agent, as well as in combination with other approved agents (including cytarabine and mitozantrone). Alvocidib is currently being evaluated in a Phase II study for the treatment of intermediate and high-risk AML in combination with standard of care agents.