FDA Approves Pomalidomide for 2 Kaposi Sarcoma Indications

The FDA approved pomalidomide (Pomalyst) for the treatment of AIDS-related Kaposi sarcoma, that has become resistant to highly active antiretroviral therapy (HAART) or for patients with Kaposi sarcoma who are HIV-negative, according to Bristol Myers Squibb, the manufacturer of the drug.

“Patients with Kaposi sarcoma have had few options to manage their disease for 2 decades,” said Diane McDowell, MD, vice president, Hematology Global Medical Affairs, Bristol Myers Squibb, in a statement. “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.”

The accelerated approval was based off overall response rate (ORR) findings from a phase I/II open-label, single-arm 12-C-0047 clinical trial. The trial included 18 HIV-positive patients and 10 HIV-negative patients who received 5 mg of pomalidomide once a day for 21 of 28-day cycles until progression or unacceptable toxicity. Patients with HIV continued HAART therapy, and patients with symptomatic pulmonary or visceral Kaposi sarcoma, history of venous or arterial thromboembolism, or procoagulant disorders were excluded from the trial.

Median time to the first response was 1.8 months. ORR was 71%, with 14% of patients achieving a complete response. Average duration of response was 12.1 months, and half of the patients who responded had a response that lasted 12 or more months.

Most common adverse events were: maculopapular rash (71%), constipation (71%), fatigue (68%), nausea (36%), diarrhea (32%), cough (29%), dyspnea (29%), peripheral edema (29%), upper respiratory tract infection (29%), muscle spasms (25%), hypothyroidism (21%), dry skin (21%) and chills (21%). Grade 3 or 4 adverse reactions included maculopapular rash (3.6%), diarrhea (3.6%) and peripheral edema (3.6%). Grade 3 or 4 laboratory abnormalities (≥5%) worsening from baseline included decreased absolute neutrophil count (50%), decreased phosphate (25%), elevated glucose (7%), and elevated creatine kinase (7%).

Eleven percent (3 patients) permanently discontinued treatment due to AEs.

Continued approval could be contingent on verification and description of clinical benefit in a confirmatory trial.

“Pomalyst has shown positive results in Kaposi sarcoma patients, regardless of their HIV status,” said Robert Yarchoan, MD, Chief of the HIV and AIDS Malignancy Branch within the Center for Cancer Research of the National Cancer Institute, 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.”