Amgen announced the European Commission (EC) has approved an expanded indication for BLINCYTO (blinatumomab) monotherapy to include adult patients with Philadelphia chromosome negative (Ph-) CD19 positive B-cell precursor acute lymphoblastic leukemia (ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1 percent. The approval was based on data from the Phase 2 BLAST study in frontline and relapsed/refractory ALL, the largest prospective trial for MRD-positive ALL ever conducted. BLINCYTO, a bispecific CD19-directed CD3 T cell engager (BiTE), is the first BiTE immunotherapy to receive regulatory approval globally.
MRD refers to the presence of cancer cells that remain detectable, despite a patient having achieved complete remission by conventional assessment. MRD is only measurable through the use of highly sensitive testing methods that detect cancer cells in the bone marrow with a sensitivity of at least one cancer cell in 10,000 cells – versus about one in 20 with a conventional microscope-based evaluation.
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"This approval represents a paradigm shift in the management of ALL in the European Union, making BLINCYTO the first and only treatment with marketing authorization to include the presence of MRD," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "We are pleased that the European Commission has seen the value BLINCYTO can bring to people living with ALL and are proud to continue to deliver on our commitment to the pursuit of breakthroughs that can transform the lives of cancer patients."
The EC approval is based on data from the Phase 2 BLAST study, which found that BLINCYTO induced a complete MRD response, or no detectable MRD, in 78 percent of patients within one treatment cycle. Safety results among MRD-positive patients were consistent with the known safety profile of BLINCYTO in relapsed or refractory B-cell precursor ALL.
"Survival rates for patients that achieve MRD-negativity are significantly higher than those for patients that remain MRD-positive, underscoring the critical importance of early testing for and eliminating residual disease in patients with ALL," said Nicola Gökbuget, M.D., principal investigator for the BLAST study and head of the German Multicenter Study Group for Adult ALL located in Frankfurt, Germany. "Data from the BLAST study demonstrated that BLINCYTO is effective in eliminating detectable residual disease and showed the potential to improve relapse-free survival in this patient population. Today's approval provides physicians across Europe with a much-needed treatment option that can potentially help prevent a relapse, which is associated with a very unfortunate prognosis."
Approval via the centralized procedure allows for obtaining a marketing authorization from the EC, which is valid in all European Union (EU) and European Economic Area (EEA)-European Free Trade Association (EFTA) states (Norway, Iceland and Liechtenstein).
In March 2018, the U.S. Food and Drug Administration (FDA) approved BLINCYTO for the treatment of adults and children with B-cell precursor ALL in first or second complete remission with MRD greater than or equal to 0.1 percent.