Genzyme Corporation (Nasdaq: GENZ) announced today that the FDA’s Oncologic Drugs Advisory Committee voted 9 to 3 that a randomized, controlled trial is needed to support the currently proposed label expansion for Clolar® (clofarabine) in adult myeloid leukemia (AML). The committee found that the single-arm clinical study results submitted to support the label expansion showed Clolar was an active agent in acute AML patients, but concluded that a randomized clinical trial should be necessary to better interpret Clolar’s efficacy and safety in the proposed patient population.
“I am happy that the three panel members who have significant experience in treating older adults with AML recognized the value of clofarabine in these patients and did not see the need for a randomized trial,” said Harry P. Erba, M.D., Ph.D., University of Michigan, one of the co-principal investigators of the CLASSIC II study.
“The panel indicated that it wasn’t an easy decision to vote for a randomized trial as Clolar was clearly active in these patients,” said Mark Enyedy, president of Genzyme Oncology and Multiple Sclerosis. “The requirement for a randomized control trial as a standard of evidence was a major focus of the discussion. We remain committed to the clinical development of Clolar in this patient population with high unmet medical need.”
Genzyme is conducting a randomized Phase 3 trial comparing clofarabine in combination with cytarabine to cytarabine alone in relapsed and refractory adult AML patients 55 years old or older. The trial continues to exceed patient accrual expectations, and results are expected in 2011. Clofarabine is also being investigated in clinical trials by most of the leading AML experts and major cooperative leukemia investigation groups in the United States and Europe.
The CLASSIC II study, discussed at the ODAC today, analyzed 112 adult AML patients aged 60 years and older with one or more unfavorable prognostic factors, including age 70 years or older, an antecedent hematologic disorder (AHD), poor performance status, or intermediate or unfavorable cytogenetics. As reported in peer-reviewed literature, and discussed by a separate panel of AML experts who recommended the trial, these risk factors predict poor outcomes in older patients with conventional induction therapy.