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Cancer Drug Information

  • Updated: 07/03/2013

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FDA Approval for Pomalidomide

Brand name(s): Pomalyst®

  • Approved for multiple myeloma after two prior treatments.

Full prescribing information is available, including clinical trial information, safety, dosing, drug–drug interactions, and contraindications.

On February 8, 2013, the Food and Drug Administration (FDA) granted accelerated approval to pomalidomide (Pomalyst® capsules, made by 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 did not respond to the last myeloma therapy. Patients in the treatment groups received either pomalidomide alone or pomalidomide plus low-dose dexamethasone.

The efficacy results demonstrated an overall response rate of 7 percent in patients treated with pomalidomide alone, and 29 percent in those treated with pomalidomide plus low-dose dexamethasone. The median response duration was not evaluable for patients treated with pomalidomide alone. Median response duration was 7.4 months for patients treated with 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 that alerts patients and health care professionals that the drug can cause embryo–fetal toxicity and venous thromboembolism.

Because of this embryo-fetal toxicity, 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 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. Female patients who are not pregnant but could become pregnant must comply with the pregnancy testing and contraception requirements. Male patients must also comply with contraception requirements. Pharmacies must be certified with the Pomalyst REMS Program, and 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 CC-4047-MM-007, a randomized phase III clinical trial comparing pomalidomide,bortezomib, and low-dose dexamethasone with 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. 

This summary was provided by Richard Pazdur, M.D., director of the FDA's Division of Oncology Drug Products, or Patricia Keegan, M.D., director of the FDA's Division of Clinical Trials Design and Analysis.

The FDA is the division of the U.S. Department of Health and Human Services charged with ensuring the safety and effectiveness of new drugs and other products. (See "Learn How Drugs and Devices Get Approved.") The FDA's mission is to promote and protect the public health by helping safe and effective products to reach the market in a timely way, and monitoring products for continued safety after they are in use.

For further information related to oncology drug approvals, regulatory information and other oncology resources, please refer to the FDA's Oncology Tools Web site.

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