FDA Grants Multiple Myeloma Drug Accelerated Approval

Daratumumab is a single agent for multiple myeloma in patients who previously received at least 3 lines of therapy.

The FDA today granted accelerated approval to a new therapeutic option for patients with multiple myeloma.

Daratumumab (Darzalex) was approved as a single agent for multiple myeloma in patients who previously received at least 3 lines of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent, or in patients who are double-refractory to a PI and an immunomodulatory agent.

The drug is the first monoclonal antibody to be approved for multiple myeloma therapy. It was previously granted priority review and breakthrough therapy designation.

“Targeting proteins that are found on the surface of cancer cells has led to the development of important oncology treatments,” said Richard Pazdur, MD, director of the FDA Office of Hematology and Oncology Products. “Darzalex provides another treatment option for patients with multiple myeloma who have become resistant to other therapies.”

The approval of daratumumab followed a multi-center, open-label study analyzing response rates in 106 patients with relapsed or refractory multiple myeloma administered daratumumab monotherapy.

Patients in the trial showed an objective response rate of 29% (95% CI: 21-39%) with a median response duration of 7.4 months (range: 1.2 to 13.1+ months).

The recommended dose and schedule for daratumumab is 16 mg/kg once per week for 8 weeks, then once bi-weekly for 16 weeks, then once every 4 weeks until disease progression.

The safety of daratumumab was evaluated in 156 patients receiving the proposed dose and schedule.

The most commonly reported side effects were infusion reactions, fatigue, nausea, back pain, pyrexia, cough, and upper respiratory tract infection.

Daratumumab was found to interfere blood bank crossmatching, specifically with Indirect Antiglobulin Tests, as a result, should a blood transfusion become necessary caregivers should inform the blood bank a patient was treated with daratumumab.

Janssen is still required, however, to conduct a multicenter, randomized trial to establish the superiority of daratumumab over standard therapy to verify and describe the drug’s clinical benefit of as a condition of approval.

There are still several ongoing multicenter, randomized trials in patients with multiple myeloma to evaluate a primary endpoint of progression-free survival.

"This is an important day for patients in the United States with double refractory multiple myeloma, who will now have Darzalex as a new treatment option for this incurable disease," said Jan van de Winkel, PhD, chief executive officer of Genmab. "The successful approval of Darzalex is the culmination of many years of hard work, perseverance and collaboration on the part of clinical study investigators, Genmab employees and our colleagues at Janssen."