FDA Panel Endorses J&J's Darzalex Faspro for Smouldering Multiple Myeloma

Johnson & Johnson (J&J) recently announced that a panel of experts from the US Food and Drug Administration (FDA) has recommended the approval of Darzalex Faspro, a combination of daratumumab and hyaluronidase-fihj, for the treatment of smouldering multiple myeloma (SMM). SMM is recognized as an early stage of the blood cancer multiple myeloma (MM), which primarily affects plasma cells, a type of white blood cell. This recommendation by the Oncologic Drugs Advisory Committee (ODAC) involved a vote where six members supported the benefit-risk profile of this subcutaneous, CD38-targeting antibody as a sole treatment option for adults with high-risk SMM, while two members opposed.

The significance of this recommendation comes against the backdrop of an estimated 36,000 new cases of multiple myeloma expected to be diagnosed in the United States this year, according to statistics from the American Cancer Society. SMM represents about 15% of these new cases. Typically, SMM isn't actively treated until it progresses to active multiple myeloma. However, emerging research suggests that patients who are at high risk of developing active MM may gain from early intervention.

Darzalex Faspro, which incorporates Halozyme’s ENHANZE drug delivery technology, is already approved in the United States for nine different multiple myeloma indications. These include four initial treatment uses for newly diagnosed patients, irrespective of their eligibility for transplants. If approved for the new indication, Darzalex Faspro would become the first treatment aimed at potentially delaying or even preventing the transition to active MM.

J&J's application to expand the use of Darzalex Faspro is backed by data from the phase 3 AQUILA trial. This study involved 390 patients with high-risk SMM and compared the effects of J&J’s medication against active monitoring. The results, with a median follow-up period of 65.2 months, revealed that the median progression-free survival (PFS) had not been reached in patients receiving subcutaneous Darzalex, unlike the 41.5 months observed in those under active monitoring. Furthermore, the estimated 60-month PFS rate was significantly higher in the Darzalex group, at 63.1%, compared to 40.8% in the monitoring group.

Sen Zhuang, vice president of oncology clinical research at J&J Innovative Medicine, highlighted the importance of this early intervention strategy. He stated that addressing high-risk SMM proactively showcased a reduction in the risk of disease progression or mortality. Zhuang emphasized that the approach taken in the AQUILA trial exemplifies J&J's aim to tackle cancer early, offering a platform to manage the disease before it develops into a more aggressive form.

The FDA will consider the ODAC's recommendation as it deliberates on the final approval decision for Darzalex Faspro in this new treatment setting. If the FDA agrees with the panel’s positive assessment, it could mark a significant advancement in the management of smouldering multiple myeloma, offering hope for delaying or preventing the progression to active disease in high-risk patients.