On February 8, 2013, the U. S. Food and Drug Administration granted accelerated approval to pomalidomide (POMALYST capsules, Celgene Corporation) for the treatment of 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.
The approval was based on the results of clinical trial CC-4047-MM-002; a multicenter, randomized, open-label study in 221 patients with relapsed and refractory multiple myeloma who had previously received lenalidomide and bortezomib and were refractory to the last myeloma therapy. The treatment arms were pomalidomide alone or pomalidomide plus low-dose dexamethasone.
The efficacy results demonstrated an overall response rate of 7% in patients treated with pomalidomide alone, and 29% in those treated with pomalidomide plus low-dose dexamethasone. The median response duration was not evaluable in the pomalidomide alone arm and was 7.4 months in the pomalidomide plus low-dose dexamethasone arm.
The most common side effects reported in the clinical trial include fatigue and asthenia, neutropenia, anemia, constipation, nausea, diarrhea, dyspnea, upper respiratory tract infections, back pain, and pyrexia. Pomalidomide is approved with a Boxed Warning alerting patients and healthcare professionals that the drug can cause embryo-fetal toxicity and venous thromboembolism.
Because of this embryo-fetal risk, pomalidomide is available only through a restricted distribution program called the POMALYST Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the POMALYSTREMS Program by enrolling and complying with the REMS requirements. Patients must sign a Patient-Physician Agreement Form and comply with the REMS requirements. Female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements. Males must comply with contraception requirements. Pharmacies must be certified with the POMALYSTREMS Program, must only dispense to patients who are authorized to receive pomalidomide and comply with REMS requirements.
As a condition of this accelerated approval, FDA will require submission of the results of clinical trial CC-4047-MM-007, a randomized clinical trial of pomalidomide added to bortezomib and low-dose dexamethasone compared to bortezomib plus low-dose dexamethasone in patients with previously-treated multiple myeloma.
The recommended dose and schedule for pomalidomide is 4 mg taken orally on days 1-21 of repeated 28-day cycles. Cycles are repeated until disease progression.
Full prescribing information is available at:
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by completing a form online at http://www.fda.gov/medwatch/report.htm, by faxing (1-800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088).