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The FDA 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.

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The news came just a week after the FDA had granted the designation to another AML drug, Volasertib (OT 5/10/14 issue).


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.


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.