EU panel backs Darzalex for high-risk smoldering multiple myeloma
CHMP opinion based on positive results from Phase 3 trial
A European Medicines Agency (EMA) committee recommended that Darzalex (daratumumab) given as an under-the-skin injection be approved for people with smoldering multiple myeloma (SMM) who are at high risk of developing overt multiple myeloma.
SMM refers to an early, asymptomatic stage of multiple myeloma, characterized by abnormal plasma cells that have not yet caused symptoms but carry a risk of progressing to active disease.
Standard care for SMM involves waiting and monitoring for signs of active cancer, but clinical trials have indicated that early treatment with Darzalex may lead to better outcomes than the traditional, watch-and-wait approach.
According to Johnson & Johnson, the company that markets Darzalex, this is the first time that the EMA’s Medicinal Products for Human Use (CHMP) has recommended approval of a therapy for high-risk SMM. The recommendation will be reviewed by the European Commission, which has final say over therapy approvals in the European Union. The commission isn’t required to abide by the CHMP’s recommendations, but it almost always does.
“The positive recommendation from the CHMP marks an important step towards addressing the needs of people living with high-risk smouldering multiple myeloma,” Ester in’t Groen, therapeutic area head for hematology in Europe, the Middle East and Africa at Johnson & Johnson Innovative Medicine, said in a company press release.
FDA panel recommendation
The CHMP’s recommendation comes two months after a U.S. Food and Drug Administration (FDA) committee voted in favor of Darzalex for high-risk SMM.
Darzalex is an antibody-based therapy that targets CD38, a protein expressed by myeloma cells. It may be given by infusion into the bloodstream or injection under the skin; the under-the-skin formulation is marketed in the U.S. as Darzalex Faspro. Various regimens containing Darzalex are approved in Europe and the U.S. to treat both newly diagnosed myeloma and myeloma that has failed to respond to other therapies.
Jordan Schecter, MD, vice president and disease area leader for multiple myeloma at Johnson & Johnson Innovative Medicine, said the therapy “has become a foundational treatment across all stages of multiple myeloma.”
“We are on a mission to continue to evolve the treatment paradigm to reach those with high-risk smouldering multiple myeloma who may benefit from proactive earlier intervention,” Schecter said. “Today’s positive CHMP opinion marks positive scientific progress towards this goal and reinforces our vision of eliminating multiple myeloma.”
The CHMP’s opinion was based on data from a Phase 3 clinical trial called AQUILA (NCT03301220). The study enrolled nearly 400 people with high-risk SMM who were randomly assigned to receive Darzalex or to standard, watch-and-wait care. After about five years of follow-up, significantly more patients given Darzalex were alive and free from disease progression — 63.1% vs. 40.8%, which works out to a more than 50% lower risk of death or disease progression with Darzalex.
“Early disease intervention with [Darzalex] has the potential to reduce the risk of progression to active multiple myeloma or death by 51 percent for patients with high-risk disease,” in’t Groen said. “Pending European Commission approval, patients and physicians will have an option to treat high-risk smouldering multiple myeloma, with the aim to intercept this complex blood cancer before it develops into active disease and importantly, before end-organ damage occurs.”
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