#FDA Approval #DARZALEX FASPRO® #smoldering myeloma

DARZALEX FASPRO®: A New Hope for High-Risk Smoldering Multiple Myeloma

On November 6, 2025, Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) approved DARZALEX FASPRO®, a groundbreaking treatment for patients with high-risk smoldering multiple myeloma (HR-SMM). This innovative therapy is the first of its kind, enabling healthcare providers to intervene earlier in the course of the disease, which could significantly impact patient outcomes.

The Significance of the Approval

The approval of DARZALEX FASPRO® is a pivotal moment for the over 36,000 Americans diagnosed with multiple myeloma annually, particularly for the estimated 15% diagnosed with the smoldering variant. Smoldering multiple myeloma (SMM) is an asymptomatic stage of the disease characterized by the presence of malignant plasma cells in the bone marrow. Approximately 50% of patients with HR-SMM may progress to active multiple myeloma within two years, making timely intervention crucial.

The FDA's decision was based on the findings of the Phase 3 AQUILA study, a comprehensive trial that compared the efficacy and safety of DARZALEX FASPRO® with traditional active monitoring. Results showed that DARZALEX FASPRO® reduced the risk of progression to active multiple myeloma or death by an impressive 51%. This suggests that the drug not only delays progression but also provides a viable treatment option where none previously existed.

Insights from the AQUILA Study

The AQUILA study involved a substantial cohort of HR-SMM patients and was among the largest Phase 3 trials in this area. After a median follow-up of 65.2 months, 63.1% of patients treated with DARZALEX FASPRO® had not progressed to active myeloma at the five-year mark, compared to just 40.7% in the active monitoring group.

In addition to its primary endpoint of progression-free survival, patients receiving DARZALEX FASPRO® exhibited a 63.4% response rate, contrasting sharply with the mere 2.0% observed in those under active monitoring: a testament to the treatment's effectiveness. The median time before patients experienced a treatment for active multiple myeloma was also notably longer for those on DARZALEX FASPRO®, further solidifying its position as a cornerstone in the therapeutic landscape of multiple myeloma.

Expert Opinions and Future Directions

Leading professionals in hematology have expressed enthusiasm regarding this treatment advancement. Dr. Peter Voorhees of Atrium Health noted that patients with high-risk SMM previously faced a limited