FDA Converts Blincyto to Full Approval for Minimal Residual Disease in B-Cell ALL

The FDA has converted the accelerated approval of Blincyto (blinatumomab) to a full approval to treat adults and pediatric patients with CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL). It is indicated for patients in first or second remission with minimal residual disease (MRD).

B-cell precursor acute lymphoblastic leukemia is a fast-growing cancer in which many B-cell lymphoblasts are found in the bone marrow and blood. The American Cancer Society estimates that in 2023 there will be about 6,540 new cases of ALL and about 1,390 deaths.

Developed by Amgen, Blincyto is a BiTE (bispecific T-cell engager) immuno-oncology therapy that targets CD19 surface antigens on B cells that was granted accelerated approval in 2018 for patients with minimal residual disease. Its current wholesale acquisition cost price is $4,900.15 per vial, according to Amgen spokesperson. “The cost reflects the significant clinical, economic and humanistic value of the product to patients and the healthcare system, for an ultra-orphan population with a serious illness,” the spokesperson told Formulary Watch. The company offers copay assistance for $0 out-of-pocket for each dose through Amgen SupportPlus.

BiTE molecules fight cancer by helping the body's immune system detect and target malignant cells by engaging T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. By bringing T cells near cancer cells, the T cells can inject toxins and trigger cancer cell death.

“In a phase 2 study, roughly 80% of adult patients treated with blinatumomab experienced a complete MRD response,” principal investigator Elias Jabbour, M.D., department of leukemia, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center in Houston, said in a press release.

The full approval is based on additional data from two phase 3 studies. These studies found that in children, the adverse reactions were similar in type to those seen in adult patients with MRD-positive ALL, and no differences in safety were observed between the different pediatric age subgroups.

Blincyto is also approved to treat adult and pediatric patients with relapsed or refractory CD19-positive B-cell precursor acute lymphoblastic leukemia.

Amgen continues development of Amgen Blincyto with studies aimed at treating patients with MRD-negative B-ALL, as well as the development of a subcutaneous formulation.