FDA Approves Pomalyst for AIDs-Related or HIV-Negative Kaposi Sarcoma

The Food and Drug Administration (FDA) approved Pomalyst (pomalidomide) to treat patients with AIDS-related Kaposi sarcoma whose disease has become resistant to highly active antiretroviral therapy or in patients with Kaposi sarcoma who are HIV-negative — the first new option for this patient population in more than 20 years.

“Patients with Kaposi sarcoma have had few options to manage their disease for two decades,” Dr. Diane McDowell, vice president of Hematology Global Medical Affairs at Bristol Myers Squibb, the agent’s manufacturer, said in a press release. “We’re excited that the additional research into Pomalyst in this rare disease area has resulted in our ability to provide a much-needed oral treatment option for patients.”

Moreover, Pomalyst is the only oral agent now approved for Karposi Sarcoma.

The FDA granted the agent accelerated approval based on overall response rates observed in a phase 1/2 open label, single-arm clinical trial designed to evaluate the safety, pharmacokinetics and efficacy of Pomalyst in 28 patients with HIV-positive and HIV-negative symptomatic Kaposi sarcoma, the majority of whom had advanced disease.

The overall response rate was 71% with four patients experiencing a complete response and 16/28 achieving a partial response. The median duration of response for all patients was 12.1 months, and half of the patients who responded maintained a response at more than 12 months.

Side effects included decreased white blood cells; elevated glucose; rash; constipation; fatigue; decreased hemoglobin, platelets, phosphate, albumin or calcium; increased alanine aminotransferase (an enzyme found in the liver); nausea; and diarrhea.

“Pomalyst has shown positive results in Kaposi sarcoma patients, regardless of their HIV status,” Dr. Robert Yarchoan, chief of the HIV and AIDS Malignancy Branch within the Center for Cancer Research of the National Cancer Institute, said in the release. “Also, it provides a therapy that is taken orally and works by a different mechanism of action than the cytotoxic chemotherapy drugs generally used to treat Kaposi sarcoma.”

Continued approval may depend on verification and description of clinical benefit in a confirmatory trial.

Bristol Myers Squibb noted in its release that patients with AIDS-related Kaposi sarcoma should continue highly active antiretroviral therapy for their HIV, as recommended by their physician.