A new oral agent is safe and effective in treating relapsed and treatment-resistant multiple myeloma, according to a recently reported, multicenter, phase II study. The agent pomalidomide is a new immunomodulatory drug under review by the FDA.

The multicenter research team evaluated 113 patients with multiple myeloma who had received at least two prior therapies and had subsequent disease progression. The study sought to determine the safety and efficacy of pomalidomide.

“When multiple myeloma no longer responds to two major classes of drugs, the average patient survival is only 9 months and progression-free survival only 5 months, representing a significant unmet need for patients with advanced disease,” said Sundar Jagannath, MD, of The Tisch Cancer Institute at The Mount Sinai Medical Center and first author of the trial. “Pomalidomide represents an exciting development for our patients.”

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Patients were administered 4 mg/day of pomalidomide plus 40 mg/week of dexamethasone for 21 days of a 28-day cycle. Progression-free survival was 4.6 months and overall survival was 16.5 months.

A subanalysis of the patients based on age was conducted. Progression-free survival was 4.7 months in patients younger than 65 years and 3.7 months in people older than 65 years. Overall survival was 19.7 months in patients younger than 65 years and 11.8 months in patients older than 65 years.

“We have made great strides in prolonging the lives of people with multiple myeloma, increasing overall survival from 3 years to as high as 7 years in less than a decade,” said Jagannath. “These results show that pomalidomide in combination with dexamethasone is a promising new option to extend survival even longer, including in older patients.”

Pomalidomide was well-tolerated, with patients experiencing side effects common to this drug class, including low WBC count, anemia, pneumonia, and low blood platelet count.

The FDA is expected to decide whether to approve pomalidomide in early 2013.

This study was reported at the American Society of Hematology annual meeting, held December 8-11, 2012, in Atlanta, Georgia.