Besponsa Approved for Acute Lymphoblastic Leukemia

FDA approves inotuzumab ozogamicin for relapsed or refractory B-cell precursor ALL.

Today, the FDA approved inotuzumab ozogamicin (Besponsa) for the treatment of patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), according to a press release from the agency.

The approval was based on positive findings from the INO-VATE ALL clinical trial, which included 326 patients with Philadelphia chromosome-negative or Philadelphia chromosome-positive relapsed or refractory B-cell precursor ALL.

Patients had ≥5% bone marrow blasts and underwent 1 or 2 previous chemotherapy treatments for ALL. Patients with Philadelphia chromosome-positive disease were required to have failed therapy with at least 1 tyrosine kinase inhibitor and chemotherapy, according to the release.

Patients were randomized to receive treatment with inotuzumab ozogamicin or chemotherapy.

Of the randomized patients treated with inotuzumab ozogamicin, 35.8% experienced complete remission (CR) with a median of 8 months. Additionally, 89.7% of these patients experienced minimal residual disease (MRD)-negativity, according to the study.

In contrast, only 17.4% of patients treated with chemotherapy experienced CR with a median of 4.9 months. The investigators reported that 31.6% of chemotherapy-treated patients achieved MRD-negativity.

The most common adverse reactions included thrombocytopenia, neutropenia, infection, anemia, leukopenia, fatigue, hemorrhage, pyrexia, nausea, headache, febrile neutropenia, increased transaminases, abdominal pain, increased gamma-glutamyltransferase, and hyperbilirubinemia, according to the FDA.

The most common adverse reactions that lead to discontinuation included infection, thrombocytopenia, hyperbilirubinemia, increased transaminases, and hemorrhage.

The recommended dose of inotuzumab ozogamicin for all patients is 1.8-mg/m2 per cycle, administered as 3 divided doses on day 1 (0.8-mg/m2), day 8 (0.5-mg/m2), and day 15 (0.5-mg/m2) for the first cycle, according to the release.

Inotuzumab ozogamicin was previously granted orphan drug, breakthrough therapy, and priority review designations for the treatment of ALL.