April 23, 2014
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FDA grants orphan drug status to Alvocidib for AML

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Tolero Pharmaceuticals Inc. today announced that the FDA has granted orphan drug designation for Alvocidib, its cyclin-dependent kinase small molecule inhibitor, for the treatment of patients with acute myeloid leukemia.

“The granting of orphan drug designation by the FDA for Alvocidib in acute myeloid leukemia is a very important milestone in the development program for Alvocidib and emphasizes our view that this is an important disease area of great unmet medical need,” David J. Bearss, PhD, CEO at Tolero Pharmaceuticals, said in a press release.

Alvocidib is currently being tested in patients with intermediate- or high-risk AML — determined by a profile of clinical, cytogenetic and molecular features — as these individuals typically have a poor prognosis due to limited treatment options.

Alvocidib has been evaluated in multiple phase 2 clinical trials including approximately 400 patients with both relapsed/refractory or front-line, previously untreated intermediate- and high-risk AML. In these trials, Alvocidib has been evaluated as a single agent as well as combined with approved agents such as cytarabine and mitozantrone.

Alvocidib also is currently being examined in a phase 2 study for the treatment of intermediate- and high-risk AML combined with standard of care agents.

“Tolero Pharmaceuticals is pleased with the results that Alvocidib has demonstrated in patients with AML, and we are actively pursuing the development of Alvocidib for intermediate- and high-risk AML patients who desperately need new options for the treatment of their disease,” Bearss said. “We look forward to sharing detailed study results of Alvocidib that support further development of the compound at an upcoming medical meeting this year.”

Orphan drug designation is granted by the FDA Office of Orphan Products Development to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States.