FDA Grants Accelerated Approval to New Drug Class for Multiple Myeloma Treatment

The U.S. Food and Drug Administration granted accelerated approval on August 13 to iberdomide, marketed as Zenbexus by Bristol-Myers Squibb, in combination with daratumumab, hyaluronidase-fihj and dexamethasone for adults with multiple myeloma who have already received at least one prior line of therapy including a proteasome inhibitor and an immunomodulatory agent. The combination, referred to as IberDd, represents the first approval for an entirely new class of medication known as a cereblon E3 ligase modulator, or CELMoD.

The approval was based on results from the EXCALIBER-RRMM trial, a randomized, multicenter, open-label study of adults with relapsed or refractory multiple myeloma who had received one or two prior lines of treatment. A key measure of the drug's effectiveness, an MRD-negative complete response, occurred in 41 percent of patients receiving the new IberDd combination, compared with 21 percent of patients on the older DVd regimen, a difference judged highly statistically significant.

The review was conducted under Project Orbis, an FDA Oncology Center of Excellence initiative that allows simultaneous review of cancer drugs by regulators in multiple countries; for this application, the FDA worked alongside Switzerland's Swissmedic. Multiple myeloma is a cancer of plasma cells in the bone marrow that, despite treatment advances in recent years, remains largely incurable, making new drug classes with distinct mechanisms of action particularly significant for patients who have exhausted standard treatment options.