FDA and EMA Accept Regulatory Submissions for Chronic Myeloid Leukemia Treatment

Mathew Shanley

Based on positive results from a Phase 3 head-to-head trial, the U.S. Food and Drug Administration (FDA) and European Medicines Association (EMA) have accepted the supplemental New Drug Application (sNDA) for Bosulif (bosutinib).

If approved, the sNDA would expand the approved usage of Bosulif to include patients with newly diagnosed chronic phase Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML). In 2012, the drug was approved by the FDA for the treatment of adult patients with Ph+ CML with resistance or intolerance to prior therapy.

With the Priority Review status, the FDA review time of the drug will be accelerated from 10 months to 6, meaning that the Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is in December 2017.

CML is a rare cancer that originates in the blood-generating cells of the bone marrow, and eventually invades the blood. CML accounts for about 10% of all leukemias and approximately 48,000 people in the United States are currently living with the condition. The American Cancer Society estimates that 9,000 new CML cases will be diagnosed in 2017.

The submissions are based on results of the Phase 3 Bosutinib trial in the First Line Chronic Myelogenous Leukemia Treatment (BFORE) study, conducted in conjunction by Pfizer and Avillion, that enrolled 536 patients with chronic Ph+ CML randomized to receive Bosulif (400 mg) or imatinib, the current standard of care for these patients. The primary endpoint in the study was met when it was seen that after 12 months of treatment, Bosulif was associated with a significantly higher rate of patients achieving major molecular response (MMR) than imatinib.

Currently, Bosulif is approved to be administered at 500 mg daily for patients who are resistant or intolerant to tyrosine kinase inhibitor (TKI) therapy. Adverse events (AE) in the study were consistent with the already-known safety profile for Bosulif. The new indication sought will be for a dosage of 400 mg per day.

“As physicians gained experience with Bosulif, they have come to appreciate its favorable risk-benefit profile in patients with Ph-positive CML who no longer responded to or could not tolerate prior TKI therapy,” said Mace Rothenberg, MD, Chief Development Officer, Oncology, Pfizer Global Product Development in a press release. “At the 400 mg dose, we believe that the BFORE study demonstrates a similarly favorable risk-benefit in previously untreated patients with Ph-positive CML. We look forward to working with the FDA in our efforts to expand the label for Bosulif to include this important group of patients.”

Pfizer and Avillion first entered into an exclusive agreement in 2014 with the intention of conducting the BFORE trial. If approved for this indication, Avillion will be eligible to receive milestone payments from Pfizer, while Pfizer will retain all rights to commercialize Bosulif worldwide.

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