The US Food and Drug Administration on Friday granted accelerated approval to Blincyto (blinatumomab) to treat adults and children with B-cell precursor acute lymphoblastic leukemia (ALL) who are in remission but still have minimal residual disease (MRD).
The FDA granted this application from US biotech major Amgen (Nasdaq: AMGN) priority review and it received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
“This is the first FDA-approved treatment for patients with MRD-positive ALL,” commented Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Because patients who have MRD are more likely to relapse, having a treatment option that eliminates even very low amounts of residual leukaemia cells may help keep the cancer in remission longer,” he added.
Drug came with acquisition on Micromet
Amgen gained rights to the drug as part of its nearly $1.2-billion acquisition of Micromet in 2012. Blincyto, which has an average wholesale price in the USA of around $173,000, generated full year 2017 to $175 million, up 52% on the prior year.
“Until today, no therapy has been satisfactory in eradicating MRD or approved specifically to treat this high-risk patient population,” said David Reese, senior vice president of Translational Sciences and Oncology at Amgen. “This approval not only supports the use of Blincyto earlier in the ALL treatment continuum, but represents a paradigm shift in the management of ALL.”
The side effects of Blincyto when used to treat MRD-positive B-cell precursor ALL are consistent with those seen in other uses of the drug. Common side effects include infections (bacterial and pathogen unspecified), fever (pyrexia), headache, infusion related reactions, low levels of certain blood cells (neutropenia, anemia), febrile neutropenia (neutropenia and fever) and low levels of platelets in the blood (thrombocytopenia).
Carries boxed warning
Blincyto carries a boxed warning alerting patients and health care professionals that some clinical trial participants had problems with low blood pressure and difficulty breathing (cytokine release syndrome) at the start of the first treatment, experienced a short period of difficulty with thinking (encephalopathy) or other side effects in the nervous system. Serious risks of Blincyto include infections, effects on the ability to drive and use machines, inflammation in the pancreas (pancreatitis), and preparation and administration errors – instructions for preparation and administration should closely be followed.
Blinatumomab was first approved by the FDA under accelerated approval in December 2014 for the treatment of Philadelphia chromosome (Ph)-negative relapsed or refractory positive B-cell precursor ALL. Full approval was granted in July 2017, when the indication was also expanded to include patients with Philadelphia chromosome-positive ALL
In November 2015, Blincyto was granted conditional marketing authorization in the European Union for the treatment of adults with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor ALL. Additional regulatory applications for Blincyto are underway and have been submitted to health authorities worldwide, said Amgen.