On the 19^th June 2025, the Committee for Medicinal Products for Human Use (CHMP) recommended  marketing authorisation for daratumumab for patients with high-risk smouldering myeloma.

Daratumumab is a monoclonal antibody marketed by Johnson & Johnson as Darzalex®. This medicine has been designed to recognise and bind to the CD38 protein, which is found on the surface of myeloma cells. By attaching to CD38, daratumumab kills myeloma cells. It also activates the immune system to find and kill more myeloma cells. It is currently used on its own (i.e., monotherapy) or in combination with other myeloma treatments at various stages of myeloma.

Daratumumab monotherapy will be indicated for the treatment of patients with smouldering myeloma who are at high risk of developing myeloma.

The CHMP is the European Medicines Agency’s committee responsible for scientific opinions on medicinal products for human use. Based on this opinion, marketing authorisation is expected to be granted by the European Commission in the next 60 days. This will be followed by further country level assessments, pricing and reimbursement decisions.

If approved, this will be the first authorised treatment option for high-risk smouldering myeloma patients.

The CHMP positive opinion is based on the results of the AQUILA clinical trial. This trial looked at whether subcutaneous (i.e., under the skin) injections of daratumumab could help patients with high-risk smouldering myeloma reduce the risk of disease progression, compared to simply monitoring their condition without treatment.

The trial enrolled 390 patients from 124 centres who received either subcutaneous daratumumab on a weekly basis for 2 months, then every 2 weeks for 4 months, and every 4 weeks thereafter for about three years or until disease progressing to myeloma, or did not receive treatment (i.e., observation). The results showed a significant benefit of daratumumab treatment compared to observation, especially in patients with highest risk of disease progression. Daratumumab delayed time to progression to myeloma and myeloma treatment initiation, and improved survival with 93% of patients being alive at 60 months compared to 87% in the observation group. Serious side effects were observed in 40% of patients in the daratumumab arm compared to 30% in the observation group. An increase in infections with daratumumab was reported and 7% of patients discontinued the trial because of side effects related to daratumumab. The trial is still ongoing and further results are needed to understand the long-term benefits of daratumumab compared to observation.

Watch our interview with Prof. Meletios Dimopoulos and Dr. Albert Oriol at the American Society of Hematology (ASH) Annual Meeting 2024 to learn more about the AQUILA trial and results.