European Commission Approves DARZALEX as First Licensed Treatment for High-Risk Smouldering Multiple Myeloma

Janssen-Cilag International NV, a Johnson & Johnson company, announced that the European Commission has approved a new indication for DARZALEX (daratumumab) subcutaneous (SC) formulation as monotherapy to treat adult patients with high-risk smouldering multiple myeloma (SMM). SMM is an asymptomatic, intermediate stage of multiple myeloma characterized by abnormal cells in the bone marrow, with a significant risk of progression to active disease.

Until now, treatment options for high-risk SMM were limited to active monitoring, despite half of these patients progressing to symptomatic multiple myeloma within two years. Professor Meletios A. Dimopoulos of the National and Kapodistrian University of Athens highlighted that this approval “offers the potential to change this trajectory,” enabling earlier intervention to delay disease progression and reduce irreversible organ damage.

Ester in ’t Groen, EMEA Therapeutic Area Head of Hematology at Johnson & Johnson, called the approval “an exciting step forward” addressing a longstanding unmet need, providing patients with an option to intervene before symptoms develop.

The approval is based on data from the Phase 3 AQUILA study, the largest randomized trial in high-risk SMM patients, comparing fixed-duration daratumumab SC monotherapy with active monitoring. After a median follow-up of over five years, patients treated with daratumumab SC showed a significant improvement in progression-free survival (PFS): 63.1% remained progression-free at 60 months compared to 40.8% in the monitoring group. Among high-risk patients, median PFS was not reached with daratumumab versus 22.1 months with monitoring. Overall survival was also extended, with a 5-year survival rate of 93.0% versus 86.9%.

Daratumumab SC recipients experienced a higher overall response rate (63.4% vs. 2.0%) and delayed the need for first-line multiple myeloma treatment significantly longer than those under active monitoring. The safety profile was consistent with previous daratumumab studies, with manageable adverse events and low discontinuation rates.

Dr. Jordan Schecter, Vice President and Disease Area Leader for Multiple Myeloma at Johnson & Johnson, stated, “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 earlier, significantly delaying progression and extending overall survival.”

This approval marks the first licensed treatment for high-risk smouldering multiple myeloma in Europe, providing a new therapeutic option to potentially alter the disease course before symptoms arise.