FDA Approves Expanded Indication of Multiple Myeloma Drug

The FDA yesterday approved an expanded indication for lenalidomide (Revlimid) 10-mg capsules for patients with multiple myeloma following an autologous hematopoietic stem cell transplant (auto-HSCT).

The approval makes lenalidomide the first treatment used as maintenance therapy following auto-HSCT, according to a press release from Celgene.

"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 new approval was based on positive findings from 2 large clinical trials that included more than 1000 patients administered lenalidomide maintenance therapy until disease progression or toxicity post-auto-HSCT versus no maintenance therapy, according to the release.

Both studies had a primary efficacy endpoint of progression-free survival (PFS). In the first study, investigators found that PFS was 5.7 years with lenalidomide, compared with 1.9 years with no treatment. The second study showed a median PFS of 3.9 years with lenalidomide, and 2 years with no maintenance therapy.

A descriptive analysis revealed that overall survival was 9.3 years for patients treated with lenalidomide, and 7 years with no maintenance therapy in study 1. In the second study, the investigators found that overall survival for patients treated with lenalidomide was 8.8 years, while overall survival was 7.3 years for the other cohort, according to the release.

As indicted in the prescribing information, lenalidomide can result in fetal harm, and should not be used in pregnant patients. Currently, the drug is only available through the lenalidomide REMS restricted distribution program, Celgene reported.

Across both trials, the most common adverse events associated with lenalidomide treatment were neutropenia, thrombocytopenia, leukopenia, anemia, upper respiratory tract infection, bronchitis, nasopharyngitis, cough, gastroenteritis, diarrhea, rash, fatigue, asthenia, muscle spasm, and pyrexia.

Lenalidomide plus dexamethasone was approved in June 2006 for therapy in previously treated patients with multiple myeloma. Its indication was expanded in February 2015 to treat newly diagnosed patients.

Celgene also submitted an application to the European Medicines Agency for the treatment of newly diagnosed patients after receiving an autologous stem cell transplant. The Agency’s Committee for Medicinal Products for Human Use approved the drug’s use as a maintenance therapy for these patients, according to the press release.

"In newly-diagnosed multiple myeloma, auto-HSCT remains a viable option for many patients and often provides a strong response against the disease," said Michael Pehl, president, Global Hematology and Oncology for Celgene. "By expanding the approval for lenalidomide to include post-transplant maintenance, patients have the potential to maintain those responses and, importantly, delay progression of the disease."