Amgen announced that the European Commission (EC) has granted conditional marketing authorization for BLINCYTO® (blinatumomab) for the treatment of adults with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL).
ALL is a rare and rapidly progressing cancer of the blood and bone marrow. For adults with relapsed or refractory ALL, the median overall survival is just three to five months. It is estimated that the incidence of adults with Ph- relapsed or refractory B-precursor ALL in the European Union (EU) is approximately 900 patients per year.
"We are pleased the European Commission granted conditional marketing authorization for BLINCYTO," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "BLINCYTO has demonstrated efficacy in treating relapsed or refractory ALL, a very difficult-to-treat disease for which historically patients had limited therapeutic options. This approval represents an important milestone in immunotherapy research. BLINCYTO is the first clinical validation of the BiTE® platform, a new and innovative approach that helps the body's own immune system fight cancer."
The conditional marketing authorization for BLINCYTO is based on results of two Phase 2 studies, study '211 and '206. In the pivotal '211 trial, 42.9 percent of patients achieved complete remission (CR) or CR with partial hematological recovery (CRh*) with single-agent BLINCYTO.
Study '211 evaluated BLINCYTO in an open-label, multicenter, single-arm Phase 2 study. Eligible patients were at least 18 years of age with Ph- relapsed or refractory B-precursor ALL relapsed with first remission duration of less than or equal to 12 months in first salvage, or relapsed or refractory after first salvage therapy, or relapsed within 12 months of allogeneic hematopoietic stem cell transplantation (HSCT), and had at least10 percent blasts in bone marrow.
Study '206 evaluated the safety and efficacy of BLINCYTO in an open-label, multicenter, dose-escalation Phase 2 study of 36 patients, who were at least 18 years of age with B-precursor ALL relapsed after at least induction and consolidation or having refractory disease with greater than 5 percent blasts in bone marrow, had an Eastern Cooperative Oncology Group (ECOG) performance status of at most 2, had a life expectancy of at least 12 weeks, and who did not have autologous HSCT within six weeks prior to start of treatment, allogeneic HSCT within three months prior to start of treatment, or previous treatment with BLINCYTO.
The most serious adverse reactions that occurred during BLINCYTO treatment in the pivotal '211 trial included infections, neurologic events, neutropenia/febrile neutropenia, cytokine release syndrome and tumor lysis syndrome.
"We tested BLINCYTO in ALL, the most aggressive B-cell malignancy we know, and observed a clinically meaningful remission rate," said Max S. Topp, M.D., professor, Hospital of Wuerzburg, Germany. "This is the first major advance in more than two decades for patients with this hard-to-treat cancer."
"The prognosis for adult patients with ALL who are refractory to treatment or experience relapse is poor, and BLINCYTO constitutes a new treatment option for these patients," said Herve Dombret, M.D., professor, University Paris, Hospital Saint Louis, Paris. "It is important for clinicians and patients to have more treatment options in this acute form of leukemia."
Approval from the EC grants a centralized conditional marketing authorization with unified labeling in the 28 countries that are members of the EU. Norway, Iceland and Liechtenstein, as members of the European Economic Area (EEA), will take corresponding decisions on the basis of the decision of the EC. Conditional license requires the license to be renewed every year and it will be converted to full standard license once post-licensing commitments have been fulfilled.
BLINCYTO was granted orphan drug designation by the European Medicines Agency in 2009 for the treatment of ALL.
