The U.S. Food and Drug Administration (FDA) expanded the approval of lenalidomide to include maintenance therapy for patients with multiple myeloma (MM) who have received autologous hematopoietic cell transplantation (HCT).
The approval was based on data from two studies (CALGB 100104 and IFM 2005-02) that compared lenalidomide maintenance therapy versus no maintenance in more than 1,000 patients. In both studies, rates of progression-free survival (PFS), the primary endpoint, were greater in the lenalidomide maintenance group than the no-maintenance cohort: 5.7 versus 1.9 years (hazard ratio [HR] = 0.38; 95% CI 0.28-0.5) and 3.9 versus 2 years (HR=0.53; 95% CI 0.44-0.64).
The most common hematologic adverse events (AEs) in the lenalidomide-treated arm included neutropenia, thrombocytopenia, leukopenia, and anemia. The frequency of AEs was highest in the first six months of treatment, then decreased over time or remained stable throughout treatment.
In 2006, the FDA approved lenalidomide for the treatment of patients with MM who received at least one prior therapy; in 2015, that approval was expanded to include patients with newly diagnosed MM.
Source: FDA Press Release, February 22, 2017.