FDA grants breakthrough designation to blinatumomab for Philadelphia chromosome-negative ALL

The FDA granted breakthrough therapy designation to blinatumomab, an investigational bispecific T-cell engager antibody, for treatment of adults with Philadelphia chromosome-negative relapsed/refractory B-precursor acute lymphoblastic leukemia, Amgen announced.

Blinatumomab is designed to direct the body’s cell-destroying T cells against target cells expressing CD19, a protein found on the surface of B-cell–derived leukemias and lymphomas.

The FDA used results from an ongoing phase 2 study of blinatumomab in 189 adult patients with Ph-relapsed/refractory B-precursor ALL to base the designation, which is intended to expedite development and review of drugs to treat serious or life-threatening conditions, according to a press release.

The interim results were presented at the 2014 ASCO Annual Meeting in Chicago, as well as the Congress of the European Hematology Association.

“There is a high unmet need for new medicines to treat relapsed and refractory ALL patients, who have very few treatment options,” Sean E. Harper, MD, executive vice president of research and development at Amgen, said in a press release. “The results from the phase 2 trial evaluating blinatumomab in adult patients with relapsed or refractory ALL are encouraging and provide a strong basis for a regulatory filing later this year and potential approval in this serious disease.”

Breakthrough therapy designation criteria require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

A breakthrough therapy designation conveys all of the fast track program features, more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review.