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Pomalyst (Pomalidomide) Receives FDA Nod for Advanced Multiple Myeloma

Drug accepted under agency’s accelerated approval program (Feb. 8)

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

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.

Pomalyst (pomalidomide) modulates the body’s immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment.

In July 2012, the FDA approved Kyprolis (carfilzomib, Onyx Pharmaceuticals), another multiple myeloma drug. Similar to Kyprolis, Pomalyst has been 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.

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

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

The labeling for Pomalyst (pomalidomide) includes 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. The drug can also cause blood clots.

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

Source: FDA; February 8, 2013.

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