Despite some concern over a novel ocular toxicity, the FDA granted accelerated approval to belantamab mafodotin (Blenrep) for heavily pretreated multiple myeloma following backing from an advisory panel, the first approved therapy that targets the B-cell maturation antigen (BCMA).
Belantamab mafodotin is indicated for patients that have failed a minimum of four prior anti-myeloma treatments, including an immunomodulatory drug, an anti-CD38 monoclonal antibody, and a proteasome inhibitor (PI).
Support for the approval came from the single-arm DREAMM-2 trial, which tested the anti-BCMA drug in 97 patients with relapsed or refractory disease. After failing a median of seven prior lines of treatment, 31% (97.5% CI 21-43) responded to treatment with belantamab mafodotin, with 73% of the responses lasting more than 6 months.
“While treatable, refractory multiple myeloma is a significant clinical challenge with poor outcomes for patients whose disease has become resistant to the current standard of care,” DREAMM-2 investigator Sagar Lonial, MD, of the Winship Cancer Institute of Emory University in Atlanta, said in a statement. “Due to the limited options currently available, these patients are often retreated with drugs from the same classes after they relapse, which is why the approval of Blenrep, the first anti-BCMA therapy, is significant for both patients and physicians alike.”
More than 70% of patients in the trial developed keratopathy and corneal changes. While these events resolved in most cases, some patients (2.1%) permanently discontinued treatment due to the toxicity. At a July meeting of the Oncologic Drugs Advisory Committee, however, members voted unanimously that the benefits of belantamab mafodotin outweighed the risks.
Due to the risk of ocular toxicity, FDA has included a boxed warning on the prescribing information, and the drug will be distributed through a Risk Evaluation and Mitigation Strategy (REMS). Full approval of the drug could be contingent on clinical outcomes in confirmatory trials.
Below are other highlights of advances in the management of multiple myeloma in 2020.