Darzalex approved in EU to treat smoldering multiple myeloma
Early stage disease progresses to full-blown cancer in some patients
Johnson & Johnson‘s Darzalex (daratumumab) is now the first approved treatment for high-risk smoldering multiple myeloma (SMM) in the European Union (EU).
SMM is an early stage of myeloma-like disease that, in some patients, will progress to overt multiple myeloma. Approval from the European Commission, which comes just a few weeks after a positive opinion from a committee of the European Medicines Agency, was based on clinical trial data indicating that Darzalex treatment can decrease the odds of high-risk SMM progressing to full-blown cancer. The approval specifically covers a subcutaneous, or under-the-skin, injection formulation of Darzalex.
“This new indication for daratumumab SC is an exciting step forward in addressing a [longstanding] unmet clinical need for those diagnosed with high-risk smouldering multiple myeloma and is the first time a treatment has been approved for this patient population,” 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.
Treatment targets key protein
Quote goes here.
Multiple myeloma is a cancer caused by the abnormal growth of plasma cells, a type of immune cell. In SMM, patients don’t have any symptoms, but do have abnormal plasma cells that can set the stage for myeloma to develop. Traditional care for SMM involves a watch-and-wait approach, where patients are monitored for signs of the disease progressing into true cancer, but no treatment is given until that time.
Authorization of Darzalex for SMM “means that eligible patients no longer have to live with the uncertainty or fear of waiting for progression to occur without active treatment, instead having the option to intercept the disease with therapeutic intervention,” in’t Groen said.
Darzalex works by targeting CD38, a protein expressed on the surface of myeloma cells. The therapy is available in subcutaneous and intravenous, or into-the-vein, formulations. Treatment regimens containing Darzalex have previously been approved in Europe and other regions to treat newly diagnosed myeloma, as well as myeloma that hasn’t responded to other therapies.
“Until now, there have been no approved treatment options for patients diagnosed with high-risk smouldering multiple myeloma,” said Jordan Schecter, MD, vice president and disease area leader, multiple myeloma, at Johnson & Johnson Innovative Medicine. “With today’s approval, Johnson & Johnson has an innovative therapy for every stage of the disease. We can now offer physicians and patients the option to treat with [Darzalex] earlier, significantly delaying progression and the need for more intensive, continuous therapy, as well as extending overall survival. We remain steadfast in our mission to get in front of cancer.”
Darzalex being evaluated as potential smoldering multiple myeloma in US
The Phase 3 AQUILA clinical trial (NCT03301220) enrolled nearly 400 people with high-risk SMM, who were randomly assigned to receive treatment with Darzalex or active monitoring. Results showed Darzalex significantly reduced the risk of disease progression or death: after five years of follow-up, 63.1% of patients given Darzalex were alive and free from disease progression, as compared to 40.8% of patients who received only monitoring. Safety data were in line with the known profile of Darzalex.
“Until now, the absence of approved therapies for high-risk smouldering multiple myeloma has left clinicians with limited options beyond observation, despite evidence that [50%] of this patient population progress to active multiple myeloma within two years,” said Meletios A. Dimopoulos, MD, a professor at National and Kapodistrian University of Athens School of Medicine. “The approval of [Darzalex] offers the potential to change this trajectory. By intervening earlier in the disease course, we have a meaningful opportunity to delay or prevent progression to symptomatic disease, reduce irreversible end-organ damage, and extend the window of improved patient outcomes.”
Darzalex is also being evaluated as a potential treatment for SMM in the U.S., where the subcutaneous version of the therapy is known as Darzalex Faspro. An advisory committee for the U.S. Food and Drug Administration recently voted in favor of the benefit-risk profile of Darzalex Faspro in high-risk SMM,
About the Author
