The U.S. Food and Drug Administration (FDA) has approved Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM).
HR-SMM and Darzalex Faspro
HR-SMM is an asymptomatic blood and bone marrow disorder, genetically identical to active multiple myeloma (MM). MM happens when specialized white blood cells in the immune system mutate into abnormal cells. Smoldering multiple myeloma may take years to become active MM. Current standard of care involves active monitoring to track signs of biochemical progression and end-organ damage.
Darzalex Faspro is a subcutaneous CD38-directed antibody. It was originally approved in 2020 for the treatment of MM and is now the first and only approved treatment for HR-SMM. The FDA approval is based on data from the AQUILA clinical trial (NCT03301220), a phase 3 randomized, multicenter study evaluating the safety and efficacy of Darzalex Faspro compared to active monitoring in patients with SMM.
Study Results
Results from the study demonstrated a significant improvement in the primary endpoint of progression-free survival (PFS). Darzalex Faspro was observed to reduce the risk of disease progression to active MM or death by 51% compared to active monitoring. Additionally, after a median follow-up of 65.2 months, 63.1% of patients who received the treatment had not progressed to active MM at 5 years, versus 40.7% in the active monitoring group.
Patients who received the treatment also saw a higher response rate of 63.4% compared to 2% with active monitoring. The median time to patients receiving first-line MM treatment was delayed for patients in the treatment group compared to active monitoring.
In a post hoc analysis of AQUILA, 41% of patients met the Mayo 2018 HR-SMM classification. Among these patients, median PFS was not reached in the Darzalex Faspro group and was 22.1 months in the active monitoring group.
Adverse reactions in the AQUILA clinical trial were generally consistent with previous studies of Darzalex Faspro. The most common adverse events in patients were upper respiratory tract infection, musculoskeletal pain, fatigue, diarrhea, rash, sleep disorder, sensory neuropathy, and injection site reactions.
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To learn more about SMM and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/