Daratumumab Approved for Multiple Myeloma in US

Zosia Chustecka

November 16, 2015

A novel option is now available for patients with multiple myeloma who have tried at least three other drug therapies ― the first monoclonal antibody for use in this disease, daratumumab (Darzalex, Janssen Pharmaceuticals, Inc), has now been approved in the United States.

The US Food and Drug Administration notes that safety and efficacy were demonstrated in two open-label studies. In one study, involving 106 patients receiving daratumumab, 29% experienced a complete or partial reduction in their tumor burden, which lasted for an average of 7.4 months. In the second study, involving 42 patients receiving the drug, 36% had a complete or partial reduction in their tumor burden.

Commenting on the study conducted in 106 patients, the principal investigator said earlier this year said that the responses seen with daratumumab were "unprecedented."

"What is striking about this study is that daratumumab, given as a single agent, produced responses in 29% of the patients on the study, even though 95% of those who participated had disease that was resistant to both their last proteasome inhibitor and lenalidomide [Revlimid, Celgene Corporation] or pomalidomide [Pomalyst, Celgene Corporation]–based regimen," said Peter Voorhees, MD, senior author and associate professor in the Division of Hematology and Oncology at the University of North Carolina School of Medicine, in Chapel Hill.

"These are patients who otherwise have very few, if any, good treatment options," he told Medscape Medical News. "Additionally, the responses were often durable, and just as importantly, although the patients enrolled in this study had been through many prior therapies and multiple relapses of their disease, no one had to stop treatment because of daratumumab-related side effects."

Adverse events include infusion-related reactions, fatigue, nausea, back pain, fever, and cough. Use of daratumumab may also result in lymphopenia, neutropenia, leukopenia, anemia, and thrombocytopenia, the FDA notes. It should not be used during pregnancy, and women who would like to become pregnant should use effective contraception for 3 months after stopping the drug.

Daratumumab may interfere with certain tests carried out by blood banks (such as antibody screening) for patients who need a blood transfusion, the FDA notes.

Daratumumab is a first-in-class human monoclonal antibody that binds to CD38-expressing cancer cells and induces cell death through multiple mechanisms. The product was given priority review and went through the FDA's accelerated approval program. It was granted breakthrough designation as well as orphan drug status.


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