U.S. FDA committee favors DARZALEX FASPRO for high-risk myeloma

Johnson & Johnson has announced the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) voted (6-2) in favor of the benefit-risk profile of single-agent DARZALEX FASPRO^® (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM). An application for the approval of DARZALEX FASPRO for adult patients with HR-SMM was submitted to the FDA in November 2024.

The vote highlights a pivotal moment in the care of patients most likely to develop active multiple myeloma (MM), potentially altering the course of disease and treatment. DARZALEX FASPRO is a foundational therapy in MM, and if approved in this indication, would provide a potential path for earlier intervention.

No treatments are approved specifically to treat high-risk smoldering multiple myeloma. In 2024, it was estimated that more than 35,000 people would be diagnosed with multiple myeloma in the U.S., and approximately 15% of newly diagnosed multiple myeloma are classified as smoldering. While patients designated with high-risk smoldering multiple myeloma are asymptomatic, approximately 50% are likely to develop active disease within two to three years. The current standard of care (SOC) for smoldering multiple myeloma, even those considered high-risk, is active monitoring (“Watch and Wait”) until progression, which may lead to therapeutic intervention only after the detection of end-organ damage.

“Early intervention in high-risk smoldering multiple myeloma demonstrated a reduction in the risk of progression or death,” said Sen Zhuang, M.D., Vice President, Oncology Clinical Research, Johnson & Johnson Innovative Medicine. “The proactive approach demonstrated in the AQUILA study is an example of Johnson & Johnson’s aspiration to get in front of cancer by providing a platform to treat disease before progression to active disease.”

The committee reviewed data from the AQUILA study, a Phase 3, randomized, open-label trial which evaluated the efficacy and safety of DARZALEX FASPRO versus SOC active monitoring in patients with HR-SMM. Results were initially presented at the 2024 American Society of Hematology (ASH) Annual Meeting and simultaneously published in The New England Journal of Medicine.

“High-risk smoldering multiple myeloma remains a challenging clinical conundrum with no approved therapies, and earlier intervention may delay or even prevent progression to active multiple myeloma,” said Peter Voorhees, M.D., Atrium Health / Levine Cancer Institute, Charlotte, N.C.‡ “We appreciate the balance the committee provided when assessing the risks and benefits of finite treatment at this stage and its recognition of the promise of DARZALEX FASPRO.”

The recommendation reinforces Johnson & Johnson’s vision for the future of oncology – one where early diagnosis and treatments become standard, and where science moves us closer to a world without cancer. With bold choices over time, J&J is dedicated to our mission of evolving the treatment paradigm of patients with multiple myeloma.

The ODAC is convened upon request of the FDA to review and evaluate safety and efficacy data of human drug products for use in the treatment of oncologic diseases. The committee provides non-binding recommendations based on its evaluation; however, final decisions on approval of the drug are made by the FDA.

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