FDA grants Breakthrough Therapy designation to inotuzumab ozogamicin

Pfizer’s investigational antibody-drug conjugate inotuzumab ozogamicin has received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for acute lymphoblastic leukaemia (ALL).

The Breakthrough Therapy designation was based on the results of the Phase 3 INO-VATE ALL trial, which enrolled 326 adult patients with relapsed or refractory CD22-positive ALL and compared inotuzumab ozogamicin to standard of care chemotherapy. Topline results from the trial demonstrated that the drug met its first primary endpoint of demonstrating a higher complete haematologic remission rate in adult patients with relapsed or refractory CD22-positive ALL.

Advancing therapies for patients with ALL is crucial

“Inotuzumab ozogamicin is the third Pfizer oncology medicine to be granted Breakthrough Therapy designation by the FDA, underscoring our commitment to innovative research and development that addresses significant unmet needs. Breakthrough Therapy designation will allow us to work more closely with the FDA to bring this important therapy to patients as rapidly as possible,” said Dr Mace Rothenberg, senior vice president of Clinical Development and Medical Affairs and chief medical officer for Pfizer Oncology. “Advancing therapies for patients with adult acute lymphoblastic leukaemia is crucial as only 10 percent of adults with ALL who relapse after first-line therapy survive five years or more with current treatment options.”

ALL is an aggressive type of leukaemia with high unmet need and a poor prognosis in adults. The current standard treatment is intensive, long-term chemotherapy. Only approximately 20 to 40 percent of newly diagnosed adults with ALL are cured with current treatment regimens.

Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy designation is intended to expedite the development and review of a potential new medicine if it is “intended to treat a serious or life-threatening disease and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies.” The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review.