FDA Names Darzalex ‘Breakthrough Therapy’ for Previously Treated Multiple Myeloma Patients

The U.S. Food and Drug Administration has designated Darzalex (daratumumab), in combination with standard-of-care regimens, a breakthrough therapy for people with previously treated multiple myeloma (MM), speeding its clinical development and regulatory review for these patients. The FDA designation is reserved for treatments showing early evidence in clinical studies of substantial improvement over existing therapies.

Darzalex is an intravenous infusion already approved as a multiple myeloma treatment. It consists of a human IgG1k monoclonal antibody (mAb) that strongly binds to the CD38 molecule, highly expressed on the surface of myeloma cells.

It was previously designed a breakthrough therapy for MM patients previously treated with at least three lines of therapy. The newest designation applies to the use of Darzalex combined with the immunomodulatory agent Revlimid (lenalidomide) and dexamethasone, or the proteasome inhibitor Velcade (bortezomib) and dexamethasone.

 “We are pleased that the FDA has granted a second breakthrough therapy designation to daratumumab,” Craig L. Tendler, MD, said in a press release. “This is an important recognition of the transformative potential of daratumumab and its possible benefit as a backbone therapy in combination with two of the most widely used regimens for multiple myeloma.”

The FDA’s latest designation was based on data from two Phase 3 clinical trials, CASTOR and POLLUX.

CASTOR evaluated 490 patients with relapsed or refractory multiple myeloma.  Patients were randomized to receive either daratumumab combined with subcutaneous bortezomib and dexamethasone or bortezomib and dexamethasone alone. The study met its primary endpoint of improved progression free survival (PFS).

The POLLUX  trial evaluated daratumumab in combination with lenalidomide and dexamethasone, compared to lenalidomide and dexamethasone alone, in patients with multiple myeloma who received at least one prior therapy. The results showed that adding daratumumab reduced the risk of disease progression or death in these patients.

“We look forward to working closely with the FDA throughout the review process and remain committed to exploring the full clinical benefit of this promising compound for multiple myeloma patients who are eagerly awaiting new treatment options,” Tendler said.