Health Canada has approved Onureg, an oral formulation of the chemotherapy azacitidine, as the first maintenance therapy for adults with acute myeloid leukemia (AML) who have achieved complete remission after intensive induction chemotherapy.
The approval is for people in remission with or without complete blood count recovery, who are not eligible for a stem cell transplant.
This decision follows U.S. Food and Drug Administration approval of Onureg, by Bristol Myers Squibb, for the same indication in September.
“While the majority of patients with AML achieve a complete remission with intensive chemotherapy, many remission patients will experience disease relapse, especially if they were not eligible for a stem cell transplant. Until now, there has been no established standard of care for Canadians who are in remission from AML, but are not eligible for a stem cell transplant,” Andre Schuh, MD, at the Princess Margaret Cancer Centre in Toronto, said in a press release.
“The approval of Onureg is significant because it gives transplant ineligible patients with AML in remission a new treatment option that may improve their survival,” Schuh added.
Azacitidine works by restoring the normal activity of genes that are often “turned off” by cancer cells, allowing them to proliferate and survive. While the chemotherapy agent is already available as an intravenous (into-the-bloodstream) and subcutaneous (under-the-skin) treatment, Onureg is a tablet formulation that allows for more convenient dosing, making it suitable as a maintenance therapy.
Approvals were supported by data from the QUAZAR AML-001 Phase 3 trial, in which Onureg significantly extended patients’ lives by a median of 10.2 months, compared with 4.8 months for those on a placebo.
QUAZAR AML-001 (NCT01757535) assessed the safety and effectiveness of Onureg as a maintenance therapy in AML patients in complete remission after intensive first-line induction chemotherapy, with or without consolidation chemotherapy.
A total of 472 participants, age 55 and older, ineligible for a stem cell transplant, and within four months of achieving complete remission were randomly assigned to Onureg plus best supportive care, or a placebo plus best supportive care.
The study’s main goal was to determine whether Onureg extended patients’ lives compared with a placebo. Additional (secondary) goals included relapse-free survival (the time until disease relapse or death).
Results showed that Onureg significantly extend patients’ overall survival, reducing the risk of death by 31%. Onureg also reduced the risk of relapse by 35%, and preserved health-related quality of life when compared with placebo.
“The approval of Onureg is an extension of our ongoing commitment to Canadians living with blood cancer,” said Al Reba, general manager, Bristol Myers Squibb Canada. “We are proud that this therapy will help to fill a significant need for Canadians living in remission from AML and hope that it will have a positive impact on their everyday life.”