FDA approves new multiple myeloma drug with REMS

February 12, 2013

 

FDA has approved pomalidomide (Pomalyst, Celgene) to treat patients 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.

Pomalidomide is the second drug approved within the past year to treat multiple myeloma. In July 2012, FDA approved carfilzomib (Kyprolis, Onyx Pharmaceuticals) to treat multiple myeloma.

Multiple myeloma is a form of blood cancer that primarily affects older adults and arises from plasma cells in the bone marrow. According to the National Cancer Institute, approximately 21,700 Americans are diagnosed with multiple myeloma and 10,710 die yearly from the disease.

“The approval of Pomalyst for the treatment of multiple myeloma serves as another addition to the list of immunomodulatory agents that now provide patients who are diagnosed with this form of blood cancer an alternative treatment if other agents fail to achieve the desired therapeutic response,” said Abimbola Farinde, PharmD, MS, clinical staff pharmacist at Clear Lake Regional Medical Center, in Webster, Texas. “This agent can be viewed as an option for patients who are refractory to other cancer agents and can help to increase the likelihood of achieving positive treatment responses.”

International Myeloma Foundation Chairman and Cofounder Brian G.M. Durie, MD, said in a company release: “Pomalyst truly fills an unmet need and is a key addition to the arsenal of therapies available to treat myeloma. The innovation that has led to the availability of new treatments, with several more to come, is really impressive."

Pomalidomide modulates the body’s immune system to destroy cancerous cells and inhibit their growth.

Pomalidomide’s safety and effectiveness was evaluated in a clinical trial of 221 patients with relapsed or refractory multiple myeloma. The trial was designed to measure the number of patients whose cancer completely or partially disappeared after treatment (objective response rate, or ORR). Patients were randomly assigned to receive pomalidomide alone or pomalidomide with low-dose dexamethasone, a corticosteroid.

Results showed 7.4% of patients treated with pomalidomide alone achieved ORR. The median duration of response has not yet been reached in these patients. In patients treated with pomalidomide plus low-dose dexamethasone, 29.2% achieved ORR with a 7.4-month median duration of response.

Pomalidomide carries a Boxed 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.

Common side effects include neutropenia, fatigue and weakness, anemia, constipation, diarrhea, thrombocytopenia, upper respiratory tract infections, back pain, and fever.