OREANDA-NEWS. Amgen (NASDAQ:AMGN) today announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for BLINCYTO® (blinatumomab) to include new data supporting the treatment of pediatric and adolescent patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). BLINCYTO, the first-and-only FDA-approved bispecific CD19-directed CD3 T cell engager (BiTE®) immunotherapy, is currently available under an accelerated approval in the U.S. for the treatment of Ph- relapsed or refractory B-cell precursor ALL, a rare and rapidly progressing cancer of the blood and bone marrow impacting both adults and children.1,2

ALL is the most common type of cancer in children.3 While 95 percent of children with ALL achieve a complete remission with first-line treatment, approximately 650 children in the U.S. each year will relapse or be refractory to treatment.4-6 Pediatric patients with relapsed or refractory ALL have poor long-term outcomes, with an overall survival of less than 10 percent.7 New approaches are needed to improve response rates and help certain patients meet eligibility requirements to receive an allogeneic hematopoietic stem cell transplantation (alloHSCT), the only potentially curative option for patients with relapsed or refractory disease.

"Children with relapsed or refractory ALL have very poor long-term outcomes and currently there are limited available therapies to induce remission," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "We look forward to collaborating with regulatory authorities to make BLINCYTO available to this ultra-orphan patient population with a high unmet medical need."

The sBLA is based on data from the Phase 1/2 '205 single-arm trial, which found that treatment with BLINCYTO induced complete remission in a clinically meaningful number of pediatric patients with Ph- relapsed or refractory B-cell precursor ALL. Overall, the types of serious adverse events (AEs) reported in the pediatric population are consistent with the known BLINCYTO safety profile. The FDA approved prescribing information for BLINCYTO includes a boxed warning for cytokine release syndrome and neurologic toxicities.