Rare Disease Report

FDA Approves Indication Expansion for Leukemia Therapy

DECEMBER 19, 2017
Mathew Shanley
Pfizer Inc. has announced that the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA, expanding the indication for Bosulif (bosutinib) to include patients with newly-diagnosed chronic phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML).

sNDAs for the therapy were submitted and accepted by both the FDA and the European Medicines Association (EMA) in August. In 2012, Bosulif was approved by the FDA for the treatment of adult patients with Ph+ CML with resistance or intolerance to prior therapy in 2012.

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

The approval is based on results from the BFORE (Bosutinib trial in the First Line Chronic Myelogenous Leukemia Treatment) study, a Phase 3 study which showed Bosulif administered at 400 mg was associated with a significantly higher rate of patients achieving major molecular response (MMR) at 12 months) than patients treated with imatinib – the current standard of care – at the same dose.

Complete cytogenic response (CCyR) rate by 12 months was 77.2% for patients treated with Bosulif compared to 66.4% for patients treated with imatinib.

“BOSULIF was Pfizer’s first treatment for hematologic malignancies, and has since become an important treatment option for Ph+ CML patients who are resistant or intolerant to previous therapy. This expanded indication has the potential to make an even greater impact on the lives of patients with CML,” said Liz Barrett, Global President, Pfizer Oncology in a press release. “Today’s news marks the third FDA approval for a Pfizer hematology medicine in just five months, a significant achievement that reinforces our commitment to patients living with blood cancers.”

The adverse events (AEs) seen in the trial were consistent with the known safety profile of the drug, and the most common adverse reactions in patients treated with the newly-approved therapy included diarrhea, nausea, thrombocytopenia, rash, increased alanine aminotransferase, abdominal pain, and increased aspartate aminotransferase.

“Among patients with CML today, with the various treatment options available, it is important to recognize the unique needs of each of my CML patients and prescribe treatments that best meet those needs,” said Jorge E. Cortes, M.D., The University of Texas MD Anderson Cancer Center. “The efficacy and distinct tolerability profile of BOSULIF make it an important and useful treatment option for newly diagnosed CML patients.”

Pfizer and Avillion entered into an exclusive agreement in 2014 with the intention of conducting the BFORE trial. With today's approval, Avillion is eligible to receive milestone payments from Pfizer, while Pfizer will retain all rights to commercialize Bosulif worldwide.

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