FDA approves Celgene's multiple myeloma drug
SUMMIT, N.J.—Celgene Corp.'s Pomalyst (pomalidomide) received the nod from the U.S. Food and Drug Administration this month for patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on or within 60 days of completion of the last therapy.
As Celgene notes, the FDA approval was based on response rate and clinical benefits—including improvement in survival or symptoms—still needs to be verified through further study.
In July 2012, the FDA approved Kyprolis (carfilzomib) to treat multiple myeloma, and the agency notes that similar to Kyprolis, Pomalyst is being approved under its accelerated approval program, which provides patients earlier access to promising new drugs while the company conducts additional studies to confirm the drug's clinical benefit and safe use. The therapy was also granted orphan product designation because it is intended to treat a rare disease or condition.
Provided in pill form, Pomalyst is designed to modulate the body's immune system to destroy cancerous cells and inhibit their growth.
"Pomalyst is the third drug in a class of immunomodulatory agents that includes lenalidomide and thalidomide, and is the second drug approved in the past year to treat multiple myeloma," said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "Treatment for multiple myeloma is tailored to meet individual patient's needs, and today's approval provides an additional treatment option for patients who have not responded to other drugs."
According to Celgene, supporting the approval were the results of MM-002, a Phase II, randomized, open-label study evaluating pomalidomide (4 mg once daily on days 1-21 of each 28-day cycle) plus low-dose dexamethasone (40 mg per day given only on days 1, 8, 15 and 22 of each 28-day cycle for patients 75 years or younger, or 20 mg per day given only on days 1, 8, 15 and 22 of each 28-day cycle for patients greater than 75 years of age) versus pomalidomide (4 mg once daily on days 1-21 of each 28-day cycle) alone in patients with relapsed multiple myeloma who were refractory to their last myeloma therapy and had received lenalidomide and bortezomib.
Of the 221 patients who could be evaluated for response, 29.2 percent achieved a partial response or better in the pomalidomide plus low-dose dexamethasone arm, compared to 7.4 percent in the pomalidomide-alone arm. Overall response rate was based on responses assessed by the Independent Review Adjudication Committee (IRAC) based on the European Group for Blood and Marrow Transplantation (EMBT) criteria. The median duration of response for patients in the pomalidomide plus low-dose dexamethasone arm was 7.4 months; however, the median has not yet been reached for the pomalidomide-alone arm.
Pomalyst carries a warning alerting patients and healthcare professionals that the drug should not be used in pregnant women because it can cause severe life-threatening birth defects, and that the drug can cause blood clots.
Because of Pomalyst's embryo-fetal risk, it is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) program. Prescribers must be certified with the Pomalyst REMS program by enrolling and complying with the REMS requirements. Patients must sign a patient-physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements. Pharmacies must be certified with the Pomalyst REMS program, must only dispense to patients who are authorized to receive the drug and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS.