Johnson & Johnson has announced the submission of regulatory applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval for Darzalex Faspro (daratumumab and hyaluronidase-fihj) and Darzalex subcutaneous (SC) formulation, respectively, for a new indication. The application targets the treatment of adult patients with high-risk smoldering multiple myeloma.
The submissions are supported by data from the Phase 3 AQUILA study (NCT03301220), which evaluated Darzalex Faspro as a monotherapy in this patient population. The results of the study will be presented at the 2024 American Society of Hematology (ASH) Annual Meeting.
Unmet Need in Smoldering Multiple Myeloma
Smoldering multiple myeloma is an early, asymptomatic precursor to active multiple myeloma, characterized by the presence of abnormal plasma cells in the bone marrow and elevated monoclonal protein levels. Approximately 15% of newly diagnosed multiple myeloma cases are classified as smoldering, and half of those with high-risk disease progress to active multiple myeloma within two years. Currently, the standard approach involves active monitoring until progression to active disease.
"There remains an unmet need for early interventions and treatments that are both effective and well tolerated in people living with smoldering multiple myeloma at high-risk of progressing to active multiple myeloma," said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Innovative Medicine, Johnson & Johnson.
The AQUILA Study
The AQUILA study is a randomized, multicenter Phase 3 trial investigating Darzalex Faspro versus active monitoring in 390 patients with high-risk smoldering multiple myeloma. Patients were diagnosed within the last five years and had no prior exposure to treatments for smoldering or active multiple myeloma. The primary endpoint is progression-free survival (PFS), with secondary endpoints including time to progression, overall response rate (ORR), and overall survival (OS).
Darzalex: A CD38-Directed Antibody
Darzalex (daratumumab) is the first CD38-directed antibody approved for multiple myeloma. Darzalex Faspro, a subcutaneous formulation, received FDA approval in May 2020 and is currently approved for nine indications in multiple myeloma. Daratumumab targets the CD38 protein, which is highly expressed on multiple myeloma cells, inducing cell death through various mechanisms.
Multiple myeloma is the second most common blood cancer worldwide and remains incurable. In 2024, estimates suggest over 35,000 new diagnoses and 12,000 deaths from multiple myeloma in the U.S. The five-year survival rate is approximately 59.8%.
