FDA Actions & Updates
The latest approvals, designations, and new indications from the U.S. Food and Drug Administration for oncology drugs.

Friday, February 24, 2017

Lenalidomide Approved as Multiple Myeloma Maintenance Therapy Following Auto-HSCT

The FDA has expanded the existing indication for lenalidomide 10 mg capsules to include use for patients with multiple myeloma as maintenance therapy following autologous hematopoietic stem cell transplant (auto-HSCT). The expanded indication makes lenalidomide the first and only treatment to receive FDA approval for maintenance use following auto-HSCT.

"Autologous stem cell transplant after induction therapy is part of the continuum of care for transplant-eligible multiple myeloma patients. However, most patients will still see their disease recur or progress after this treatment," said Philip McCarthy, MD, Director, Blood and Marrow Transplant Center, Department of Medicine at Roswell Park Cancer Institute. "Lenalidomide maintenance therapy, which has been shown to increase progression-free survival following autologous stem cell transplant in clinical trials can be considered a standard of care for these patients."

The approval was based on two large studies including more than 1,000 patients comparing lenalidomide maintenance therapy given until disease progression or unacceptable toxicity after auto-HSCT versus no maintenance. In both studies, the primary efficacy endpoint was progression-free survival (PFS) defined from randomization to the date of progression or death, whichever occurred first. In the most current PFS analysis, the CALGB 100104 study demonstrated a median PFS of 5.7 years (95% CI: 4.4-not estimable) versus 1.9 years (95% CI: 1.6-2.5) for no maintenance, a difference of 3.8 years (HR 0.38 [95% CI: 0.28-0.50]). The second study, IFM 2005-02, also showed a benefit with a median PFS of 3.9 years (95% CI: 3.3-4.7) versus 2 years (95% CI: 1.8-2.3) for no maintenance, a difference of 1.9 years (HR 0.53 [95% CI: 0.44-0.64]).

Individual studies were not powered for an overall survival endpoint. A descriptive analysis showed the median overall survival in CALGB 100104 was 9.3 years (95% CI: 8.5-not estimable) for patients who received lenalidomide versus 7 years (95% CI: 5.9-8.6) for no maintenance (HR 0.59 [95% CI: 0.44-0.78]). In the CALGB 100104 study, median overall survival was 8.8 years (95% CI: 7.4-not estimable) for patients who received lenalidomide versus 7.3 years (95% CI: 6.7-9.0) for no maintenance (HR 0.90 [95% CI: 0.72-1.13]).

Lenalidomide in combination with dexamethasone was previously approved in June 2006 for use in patients with multiple myeloma who have received at least one prior therapy, and the indication expanded in February 2015 to include patients newly diagnosed with multiple myeloma.