FDA
24th July 2019

Oncologic Drugs Advisory Committee Supports Pexidartinib but Not Quizartinib

The Oncologic Drugs Advisory Committee (ODAC) reviewed the new drug application (NDA) #211810.

Oncologic Drugs Advisory Committee Supports Pexidartinib but Not Quizartinib

The Oncologic Drugs Advisory Committee (ODAC) reviewed the new drug application (NDA) #211810 for perxidartinib and NDA # 212166 for quizartinib by Daiichi Sankyo on May 14th, 2019. Pexidartinib was evaluated for the treatment of symptomatic tenosynovial giant cell tumors (TGCT) in adult patients.  The intended use of quizartinib was for the treatment of Feline McDonough Sarcoma-like tyrosine kinase 3 internal tandem duplication (FTL3-ITD) positive relapsed or refractory (R/R) acute myeloid leukemia (AML).

Pexidartinib is a tyrosine kinase inhibitor (TKI) that targets the colony stimulating factor (CSF-1) receptor in the synovium of abnormal tumor cells. To demonstrate efficacy and safety, ENLIVEN, a two-part, multicenter, double-blind, randomized, placebo-controlled phase III study was conducted. Phase I and phase II clinical studies were also evaluated.

The morning meeting had 1 discussion question and 1 voting question. The voting question asked whether the benefits of pexidartinib outweigh the risks. It resulted in a positive vote of 12 YES to 3 NO. Those who disagreed were concerned about the potential for improper utilization and increased incidence of liver toxicity.

Quizartinib is a type II tyrosine kinase inhibitor of Feline McDonough Sarcoma-like tyrosine kinase 3 (FLT3) signaling and FLT3-ITD dependent cell proliferation and survival. To demonstrate the efficacy and safety there was one phase I, two phase II, and the QuANTUM-R pivotal phase III study.

The afternoon meeting had 2 discussion questions and 1 main voting question. The voting question asked if the results of QuANTUM-R properly demonstrated that the benefits outweigh the safety risks for patients with R/R FLT-ITD-positive AML. It resulted in a vote of 3 YES and 8 NO. Those in support believed that the potential risk of QT prolongation was not a major concern. However, the majority were not convinced that the benefits demonstrated in QuANTUM-R outweighed the safety risks.

The agency is not obligated to follow its panels’ recommendation, but normally does so.

While the pexidartinib status is pending, on June 21st, Daiichi Sankyo announced it received a Complete Response Letter (CRL) for Quizartinib. This letter addresses the concern and deficiencies that prior to resubmission must be resolved.

ISS has almost two decades of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact info@innovativescience.net

This post was authored by Sharon Christian. Sharon is a summer intern at ISS and an undergraduate student at Rutgers University.

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