Pfizer’s investigational inotuzumab ozogamacin has received breakthrough therapy designation from the FDA for acute lymphoblastic leukaemia (ALL).
The decision comes on the back of data from the phase III INO-VATE ALL trial involving 326 patients with relapsed or refractory ALL which showed that inotuzumab outperformed current standards of care.
The co-primary endpoint of complete remission with incomplete haematologic recovery was achieved by 80.7% for those given inotuzumab versus 33.3% for those given standard care.
Inotuzumab is an investigational antibody-drug conjugate (ADC), which includes a monoclonal antibody targeting CD22. When inotozumab binds to the CD22 antigen on malignant B-cells, it is internalised into the cell where the cytotoxic agent calicheamicin is released and destroys the cell.
Mace Rothenberg, senior VP of clinical development and medical affairs at Pfizer, said: “Inotozumab 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.
“Advancing therapies for patients with adult acute lymphoblastic leukaemia is crucial as only 10% of adults with ALL who relapse after first-line therapy survive five years or more with current treatment options.”
Inotuzumab recently failed to improve overall survival in patients with non-Hodgkin’s lymphoma, so the designation will come as good news for Pfizer now that the drug looks to be back on track for regulatory approval.
ALL affects about 6,000 Americans every year with only 20-40% of newly diagnosed adults being cured with current treatment regimens.
The FDA’s breakthrough therapy designation was launched in 2012 and is intended to expedite the development and review of medicines for life-threatening diseases where clinical evidence suggests it offers an improvement over current therapies.
Pfizer’s other oncology medications which received breakthrough therapy designation are Ibrance (palbociclib) and Xalkori (crizotinib). According to analysts, Ibrance, which was approved for breast cancer earlier this year, could achieve sales of at least $3bn.