The FDA has approved inotuzumab ozogamicin for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
"For adult patients with B-cell ALL whose cancer has not responded to initial treatment or has returned after treatment, life expectancy is typically low," said Richard Pazdur, MD, Director of the FDA's Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "These patients have few treatments available and today's approval provides a new, targeted treatment option."
The safety and efficacy of inotuzumab ozogamicin was studied in a randomized trial of 326 patients with relapsed or refractory B-cell ALL who had received one or two prior treatments. Patients were randomized to receive treatment with inotuzumab ozogamicin or an alternative chemotherapy regimen. The trial measured the percentage of patients with complete remission (CR). Of the 218 evaluated patients, 35.8 percent who received inotuzumab ozogamicin experienced CR for a median 8.0 months; of the patients who received alternative chemotherapy, 17.4 percent experienced CR for a median 4.9 months.
Common side effects of inotuzumab ozogamicin include low levels of thrombocytopenia, neutropenia/leukopenia, infection, anemia, fatigue, hemorrhage, pyrexia, nausea, headache, febrile neutropenia, increase in transaminases and/or gamma-glutamyltransferase, abdominal pain, and hyperbilirubinemia. Other serious side effects of inotuzumab ozogamicin include myelosuppression, infusion-related reactions, and problems with the heart's electrical pulses.
The FDA granted this application Priority Review and Breakthrough Therapy designations. Inotuzumab ozogamicin also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.