EC grants expanded approval for Amgen’s BLINCYTO in pediatric acute lymphoblastic leukemia treatment

In a significant development for pediatric oncology, the European Commission (EC) has approved an expanded indication for Amgen’s BLINCYTO (blinatumomab) as a monotherapy treatment for certain types of acute lymphoblastic leukemia (ALL) in children aged one year and older. This approval marks a crucial advancement in the treatment options available for this challenging medical condition.

The EC’s decision allows BLINCYTO to be used in treating pediatric patients with Philadelphia chromosome-negative (Ph-) CD19 positive B-cell precursor ALL who are either refractory or have relapsed after receiving more than two prior therapies or after an allogeneic hematopoietic stem cell transplantation. This expansion is particularly significant given the limited pharmacological options previously available beyond traditional chemotherapy, which often resulted in poor outcomes for this vulnerable patient group.

The approval was based on compelling data from the phase 1/2 ‘205 single-arm trial, which demonstrated BLINCYTO’s efficacy and safety in 93 pediatric patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. These findings underscore the drug’s potential to improve treatment outcomes in pediatric populations.

BLINCYTO, a pioneering bispecific T cell engager (BiTE) immunotherapy, functions by binding to CD19 expressed on the surface of B-lineage cells and CD3 expressed on effector T cells. This dual binding facilitates the body’s own immune response to target and destroy leukemia cells, representing a novel approach in cancer immunotherapy.

First approved in the European Union in 2015 for adults with the same leukemia type, BLINCYTO has also received full approval in the United States for both adult and pediatric patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Additionally, in April 2018, BLINCYTO was granted accelerated approval in the US for patients in first or second complete remission with minimal residual disease greater than or equal to 0.1%.

David M. Reese, Executive Vice President of Research and Development at Amgen, emphasized the importance of this approval: “This approval for BLINCYTO provides physicians across Europe with an important new immunotherapy option for these young, heavily pretreated patients, delivering on Amgen’s commitment to making a difference in the lives of cancer patients.”

The expanded EC approval of BLINCYTO offers a new horizon of hope for children suffering from acute lymphoblastic leukemia across Europe, providing a vital new treatment option that leverages the power of the body’s immune system to fight cancer. This decision is a testament to ongoing innovations in pediatric cancer treatment and Amgen’s dedication to addressing urgent medical needs in oncology.

The European Commission’s approval of BLINCYTO for pediatric use represents a significant step forward in the treatment of acute lymphoblastic leukemia. It reflects the growing importance of targeted immunotherapies in oncology, which are poised to transform treatment paradigms and significantly improve patient outcomes in cancers that have historically had limited treatment success.