FDA Approves Pomalidomide for Advanced Multiple Myeloma

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The FDA approved pomalidomide (Pomalyst) an oral immunomodulatory agent, to treat patients with multiple myeloma whose disease progressed after being treated with other cancer drugs.

Pomalidomide is intended for patients who have received at least two prior therapies, including lenalidomide (Revlimid) and bortezomib (Velcade), and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory).

“Pomalidomide is the third drug in a class of immunomodulatory agents that includes lenalidomide and thalidomide [Thalomid], and is the second drug approved in the past year to treat multiple myeloma,” said Richard Pazdur, MD, 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.”

Accelerated Approval

In July 2012, FDA approved carfilzomib (Kyprolis) to treat multiple myeloma. Similar to carfilzomib, pomalidomide is being approved under the agency’s 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.

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). Patients were randomly assigned to receive pomalidomide alone or pomalidomide with low-dose dexamethasone.

Results showed 7.4% of patients treated with pomalidomide alone achieved objective response rate. 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 objective response rate with a 7.4-month median duration of response.

Safety Data

Pomalidomide carries a Boxed Warning alerting patients and health-care 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 pomalidomide’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 a neutropenia, fatigue and weakness, anemia, constipation, diarrhea, thrombocytopenia, upper respiratory tract infections, back pain, and fever.

Pomalidomide is marketed by Celgene, based in Summit, New Jersey. ■