FDA Approves Maintenance Lenalidomide for Multiple Myeloma

Article

The FDA has expanded the approval of lenalidomide to include its use as maintenance therapy for patients with multiple myeloma following autologous stem cell transplant.

Lenalidomide is the first maintenance therapy approved for use following ASCT

The US Food and Drug Administration (FDA) has expanded the approval of lenalidomide (Revlimid) to include its use as maintenance therapy for patients with multiple myeloma following autologous stem cell transplant (ASCT).

“ASCT 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 of the Blood and Marrow Transplant Center at Roswell Park Cancer Institute in Buffalo, in a press release. “Lenalidomide maintenance therapy, which has been shown to increase progression-free survival (PFS) following ASCT in clinical trials can be considered a standard of care for these patients.”

The expanded approval was based on a pair of studies (CALGB 100104 and IFM 2005-02) that together included over 1,000 patients. Following ASCT, the studies compared maintenance therapy with lenalidomide vs placebo, with PFS as the primary endpoint.

In CALGB 100104, the median PFS in patients who received maintenance therapy was 5.7 years vs 1.9 years with placebo, for a hazard ratio (HR) of 0.38 (95% CI, 0.28–0.50).

In IFM 2005-02, a European-based study, lenalidomide maintenance yielded a median PFS of 3.9 years vs 2 years with placebo, for an HR of 0.53 (95% CI, 0.44–0.64). The studies were not powered to measure overall survival.

The most frequently reported adverse events (20% or higher in either trial) among patients receiving lenalidomide were thrombocytopenia, leukopenia, neutropenia, anemia, diarrhea, gastroenteritis, bronchitis, nasopharyngitis, upper respiratory tract infection, cough, rash, asthenia, muscle spasm, pyrexia, and fatigue. Onset of adverse events was generally highest in the first 6 months of treatment.

The most frequently reported grade 3/4 events (greater than 20%) were thrombocytopenia, leukopenia, and neutropenia.

Second primary malignancies were also more frequent in patients receiving lenalidomide maintenance (7.5% had second hematologic cancers vs 3.3% with placebo). When including solid tumors, the incidence rate of second primary cancers was 14.9% among patients receiving lenalidomide vs 8.8% among those receiving placebo. Non-melanoma skin cancers were also higher (3.9% vs 2.6%).

Recent Videos
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.