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Immunotherapy Blinatumomab Receives FDA ‘Priority Review’ Designation for Acute Lymphoblastic Leukemia

Approval decision expected in May 2015

The FDA has accepted for review a biologics license application (BLA) for the investigational bispecific T-cell engager (BiTE) antibody construct blinatumomab (Amgen). The BLA is for the treatment of adults with Philadelphia-negative (Ph-) relapsed/refractory B-precursor acute lymphoblastic leukemia (ALL), a rapidly progressive cancer of the blood and bone marrow. As part of its acceptance, the FDA granted blinatumomab a “priority review” designation, with a Prescription Drug User Fee Act (PDUFA) action date of May 19, 2015.

The submission includes data from a phase II trial of adult patients with Ph- relapsed/refractory B-precursor ALL treated with blinatumomab, which met its primary endpoint.

According to the FDA, a “priority review” designation is assigned to applications for drugs that treat serious conditions and that would, if approved, provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.

It has been estimated that more than 6,000 cases of ALL will be diagnosed in the U.S. in 2014. In adult patients with relapsed or refractory ALL, the median overall survival period is only 3 to 5 months.

BiTE antibody constructs are a type of immunotherapy being investigated for fighting cancer by helping the body’s immune system to detect and target malignant cells. The modified antibodies are designed to engage two different targets simultaneously, thereby juxtaposing T cells to cancer cells. BiTE antibody constructs help place the T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins and to trigger the cancer cell to die (apoptosis). Such antibody constructs are currently being investigated for their potential to treat a variety of cancers. Blinatumomab is an investigational BiTE antibody construct designed to direct the body’s cell-destroying T cells against target cells expressing CD19, a protein found on the surface of B-cell derived leukemias and lymphomas.

The therapy has received an “orphan drug” designation from the FDA for the treatment of ALL, chronic lymphocytic leukemia (CLL), hairy cell leukemia, prolymphocytic leukemia, and indolent B-cell lymphoma. It is also being investigated for its potential to treat pediatric relapsed/refractory ALL, relapsed/refractory Philadelphia-positive (Ph+) B-precursor ALL, minimal residual disease positive (MRD+) B-precursor ALL, and relapsed/refractory non-Hodgkin's lymphoma (NHL), including relapsed/refractory diffuse large B-cell lymphoma (DLBCL).

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