FDA Approved Besponsa for Children With Acute Lymphoblastic Leukemia

The Food and Drug Administration (FDA) has approved Besponsa (inotuzumab ozogamicin) for pediatric patients 1 year and older with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL), the agency has announced.

The efficacy of Besponsa was determined in a multicenter, single-arm, open-label study with 53 pediatric patients 1 year old and older with relapsed or refractory CD22-positive B-cell precursor ALL, according to the agency, which noted that the study evaluated two dose levels: an initial dose of 1.4 mg/m2/cycle in 12 patients and 1.8 mg/m2/cycle in 41 patients, with premedications including methylprednisolone 1 mg/kg (maximum of 50 mg), an antipyretic and an antihistamine, and patients receiving a median of 2 cycles of therapy.

Twenty-two of the 53 patents, or 42%, achieved complete response, indicating that their disease completely disappeared, with a median duration of complete response being 8.2 months.

Additionally, minimal residual disease (MRD)-negative complete response, defined as 5% or less blasts found in the bone marrow and no cancer found in the peripheral blood, occurred in 21 of 22 patients (95.5%) who had a complete response, based on flow cytometry, and 19 of 22 (86.4%) based on RQ-PCR, which is a test of the blood and bone marrow.

Besponsa was initially approved by the FDA in 2017 for the treatment of adults with relapsed or refractory ALL. According to the drug’s website, Besponsa is an antibody drug conjugate that works by targeting and binding to CD22, which is a protein found on many B-cell ALL cells. It then is internalized by the cancer cell and releases the cancer-killing component.

The most common side effects from Besponsa that occurred in 20% or more of patients were thrombocytopenia, fever, anemia, vomiting, infection, hemorrhage, neutropenia, nausea, leukopenia, febrile neutropenia, increased transaminases, abdominal pain and headache.

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