Photo courtesy of Amgen
Vials of blinatumomab powder and solution for infusion
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding marketing authorization for blinatumomab (Blincyto) to include treatment of minimal residual disease (MRD).
The CHMP has recommended approval for blinatumomab as monotherapy for adults with Philadelphia chromosome-negative, CD19-positive, B-cell precursor acute lymphoblastic leukemia (BCP-ALL) in first or second complete remission with MRD greater than or equal to 0.1%.
The CHMP had originally adopted a negative opinion on extending the use of blinatumomab to these patients. However, the committee re-examined its opinion and reversed that decision.
The CHMP has requested that Amgen, the company developing blinatumomab, provide results from ongoing studies to support the new approval.
The CHMP’s recommendations are reviewed by the European Commission (EC), which has the authority to approve medicines for use in the European Union, Norway, Iceland, and Liechtenstein.
The EC usually makes a decision within 67 days of CHMP recommendations.
Blinatumomab is already EC-approved as monotherapy for:
- Adults with Philadelphia chromosome-negative, CD19-positive, relapsed or refractory BCP-ALL.
- Pediatric patients age 1 year or older who have relapsed/refractory, Philadelphia chromosome-negative, CD19-positive BCP-ALL and have received at least two prior therapies or relapsed after allogeneic hematopoietic stem cell transplant.
CHMP’s reversal of opinion
In July, the CHMP recommended against approving blinatumomab to treat patients with MRD based on data from the BLAST trial. Results from this phase 2 trial were published in Blood in April.
The CHMP noted that, although blinatumomab produced MRD negativity in many patients in the BLAST trial, there is no strong evidence that this leads to improved survival.
Given the uncertainty, the CHMP was of the opinion that the benefits of blinatumomab do not outweigh its risks in MRD-positive BCP-ALL patients.
However, Amgen request a re-examination of the CHMP’s opinion, and the CHMP complied.
During the re-examination, the CHMP reviewed all the data and consulted a group of experts.
The experts concluded, and the CHMP agreed, that, although there is no strong evidence of improved survival, the available data indicate a good response to blinatumomab, with around 78% of patients becoming MRD-negative after treatment.
The CHMP also noted that MRD-positive patients have a high risk of relapse and few treatment options.
Therefore, the committee concluded that the benefits of blinatumomab outweigh its risks in this patient population.
The CHMP recommended granting the change to the marketing authorization but also requested that Amgen provide data from ongoing studies of blinatumomab in MRD-positive patients, once those data are available.