After a positive phase 3 trial, Johnson & Johnson hopes its Darzalex could become the first treatment option for patients with smoldering multiple myeloma, an early-stage form of the blood disorder.
J&J has filed applications with the FDA and European Medicines Agency seeking approval for subcutaneous Darzalex, also known as Darzalex Faspro, as monotherapy for high-risk smoldering multiple myeloma, the company said Friday.
Smoldering myeloma isn’t considered a cancer, but it may develop into active myeloma. Monitor-and-wait is the current standard of care for this early-stage myeloma. About half of those diagnosed with high-risk disease will progress to active myeloma within two years, according to a 2022 estimation.
“There remains an unmet need for early interventions and treatments that are both effective and well tolerated” in those high-risk patients, Yusri Elsayed, Ph.D., J&J’s oncology global therapeutic area head, said in a statement Friday.
J&J’s filing is based on results from the phase 3 AQUILA trial. Among 390 patients with high-risk smoldering multiple myeloma enrolled in the study, subcutaneous Darzalex reduced the risk of progression or death by 51% versus active monitoring, according to an abstract for the upcoming American Society of Hematology 2024 annual meeting.
The median progression-free survival time was not reached in the Darzalex group, versus 41.5 months for control. An estimated 63.1% of patients in the Darzalex arm remained alive and progression-free at five years, versus 40.8% of those in the control group.
In addition, the J&J anti-CD38 antibody also reduced the risk of needing first-line multiple myeloma treatment by 42% compared with active monitoring. As of the data cutoff, 64 patients (33%) in the Darzalex group and 102 (52%) in the monitoring group had started first-line myeloma treatment.
The drug was also well tolerated. The rate of grade 3 or 4 treatment-emergent adverse events (TEAEs) was 40.4% and 30.1% for Darzalex and control, respectively. The rates of fatal cases from TEAEs were low in both groups, at 1% and 2%, respectively.
As the leading CD38 agent, Darzalex ginned up $8.6 billion in worldwide sales in the first nine months of 2024, good for 19% growth year over year.
Sanofi’s rival drug Sarclisa is trying to catch up. In September, Sarclisa broke into first-line myeloma with an FDA nod for use in combination with bortezomib, lenalidomide and dexamethasone (VRd) in patients who are not eligible for stem cell transplant. J&J has quickly followed with its own FDA filing in September for Darzalex’s VRd combo in transplant-ineligible patients.
Back in July, J&J got the Darzalex-VRd combo approved in transplant-eligible patients with newly diagnosed myeloma. Darzalex’s label includes other combos in the first-line setting.