The use of Revlimid (lenalidomide) as a maintenance therapy for adult patients with newly diagnosed multiple myeloma who underwent autologous stem cell transplant (ASCT) has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).
The European Commission, which generally follows the CHMP recommendations, is expected to make its final decision within two months. If approved, Revlimid will be the first and only licensed maintenance therapy available for this patient population.
“Despite substantial progress made so far in multiple myeloma treatment, it remains an incurable disease,” Tuomo Pätsi, president of Celgene in Europe, the Middle East and Africa, said in a news release. “We welcome this CHMP opinion as it confirms the important role that Revlimid plays in treating multiple myeloma, extending the use of Revlimid across the disease continuum.”
Pätsi said Celgene tries to make challenging diseases such as myeloma into “manageable conditions.” The company invests more than one-third of its revenues back into research and development, he said.
Multiple myeloma is a rare and life-threatening disease that affects 39,000 new patients every year in Europe. Although ASCT, which uses the patient’s own stem cells, is sometimes part of the treatment plan for myeloma plans, more than half of patients relapse within two to three years after receiving the transplant. As a result, researchers have been working on ways to maintain the response to therapy, ultimately delaying disease progression.
Recently, two Phase 3 trials have shown that Revlimid could be a promising maintenance therapy to prolong patients’ responses to ASCT.
The CALGB 100104 study was a randomized, controlled, double-blind, multi-center trial designed to assess the effectiveness of Revlimid in 460 newly diagnosed myeloma patients after they underwent ASCT. The patients, ages 18 to 70, who achieved at least stable disease 100 days after ASCT were randomized to receive daily Revlimid maintenance or a placebo until disease progression or intolerable side effects.
The IFM 2005-02 trial was a randomized, controlled, double-blind, multi-center study that assessed the efficacy of a two-month consolidation treatment with Revlimid followed by Revlimid maintenance therapy. The trial enrolled newly diagnosed myeloma patients younger than 65. Those who had no signs of disease progression within six months of undergoing stem cell transplant were then randomized to receive a Revlimid consolidation therapy (25 mg per day in the first three weeks of a 28-day cycle) followed by Revlimid maintenance (10 mg per day for three months, then 15 mg per day), or a placebo until disease progression or unacceptable toxicity.
In both studies, Revlimid maintenance following ASCT significantly increased progression-free survival and decreased the risk of death in patients with multiple myeloma, which supported the CHMP recommendation.
The studies also showed that patients receiving Revlimid were at a higher risk of developing second cancers compared to the placebo group. Nonetheless, the CHMP opinion confirmed that the benefits outweighed the potential risks of this expanded indication.