FDA approves Celgene's multiple myeloma drug
The compound, Pomalyst, will only be available in the United States through Pomalyst REMS, a restricted distribution program
SUMMIT, N.J.—Celgene Corp.'s Pomalyst (pomalidomide)received the nod from the U.S. Food and Drug Administration this month forpatients with multiple myeloma who have received at least two prior therapies, includinglenalidomide and bortezomib, and have demonstrated disease progression on orwithin 60 days of completion of the last therapy.
As Celgene notes, the FDA approval was based onresponse rate and clinical benefits—including improvement in survival orsymptoms—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 toKyprolis, Pomalyst is being approved under its accelerated approval program, whichprovides patients earlier access to promising new drugs while the companyconducts additional studies to confirm the drug's clinical benefit and safeuse. The therapy was also granted orphan product designation because it isintended to treat a rare disease or condition.
Provided in pill form, Pomalyst is designed tomodulate the body's immune system to destroy cancerous cells and inhibit theirgrowth.
"Pomalyst is the third drug in a class ofimmunomodulatory agents that includes lenalidomide and thalidomide, and is thesecond drug approved in the past year to treat multiple myeloma," said Dr. RichardPazdur, director of the Office of Hematology and Oncology Products in FDA'sCenter for Drug Evaluation and Research. "Treatment for multiple myeloma istailored to meet individual patient's needs, and today's approval provides an additionaltreatment option for patients who have not responded to other drugs."
According to Celgene, supporting the approval werethe results of MM-002, a Phase II, randomized, open-label study evaluatingpomalidomide (4 mg once daily on days 1-21 of each 28-day cycle) plus low-dosedexamethasone (40 mg per day given only on days 1, 8, 15 and 22 of each 28-daycycle 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) alonein patients with relapsed multiple myeloma who were refractory to their lastmyeloma therapy and had received lenalidomide and bortezomib.
Of the 221 patients who could be evaluated forresponse, 29.2 percent achieved a partial response or better in thepomalidomide plus low-dose dexamethasone arm, compared to 7.4 percent in thepomalidomide-alone arm. Overall response rate was based on responses assessed bythe Independent Review Adjudication Committee (IRAC) based on the EuropeanGroup for Blood and Marrow Transplantation (EMBT) criteria. The median durationof response for patients in the pomalidomide plus low-dose dexamethasone armwas 7.4 months; however, the median has not yet been reached for the pomalidomide-alonearm.
Pomalyst carries a warning alerting patients andhealthcare professionals that the drug should not be used in pregnant womenbecause it can cause severe life-threatening birth defects, and that the drugcan cause blood clots.
Because of Pomalyst's embryo-fetal risk, it isavailable only through the Pomalyst Risk Evaluation and Mitigation Strategy(REMS) program. Prescribers must be certified with the Pomalyst REMS program byenrolling and complying with the REMS requirements. Patients must sign a patient-physicianagreement form and comply with the REMS requirements. In particular, femalepatients who are not pregnant but can become pregnant must comply with thepregnancy testing and contraception requirements, and males must comply withcontraception requirements. Pharmacies must be certified with the Pomalyst REMSprogram, must only dispense to patients who are authorized to receive the drugand must comply with REMS requirements. Both lenalidomide and thalidomide havesimilar REMS.