FDA Grants Accelerated Approval to Pomalidomide for Kaposi Sarcoma

On May 14, 2020, the US Food and Drug Administration (FDA), following failure of highly active antiretroviral therapy in adult patients with AIDS-associated HIV-negative Kaposi sarcoma, expanded the indication for pomalidomide (Pomalyst) for this population.

Pomalidomide (marketed as Pomalyst in the United States and Imnovid in the EU) is a thalidomide derivative manufactured by Celgene. It acts through antiangiogenic and immunomodulatory mechanisms to exert anticancer activity.

Pomalidomide was approved by the US Food and Drug Administration (FDA) in February 2013 for the treatment of relapsed and refractory multiple myeloma. This new approval is pomalidomide's 2nd FDA approval.

Study 12-C-0047

The effectiveness of pomalidomide in Kaposi sarcoma was investigated in Study 12-C-0047, an open-label, single-arm clinical trial conducted by the National Cancer Institute. 28 patients (18 HIV-positive and 10 HIV-negative) received oral pomalidomide 5 mg once daily on days 1-21 of each 28-day cycle until disease progression or unacceptable side effects. All HIV-positive patients continued highly active antiretroviral therapy.

The main efficacy outcome measure was the overall response rate, including complete response, clinical complete response and partial response. Response was assessed by the investigator according to the AIDS Clinical Trials Group Oncology Committee response criteria for Kaposi sarcoma.

Results

• Among the 18 HIV-positive patients, the overall response rate was 67% (95% CI: 41, 87), and the mean duration of response was 12.5 months (95% CI: 6.5, 24.9).
• Among the 10 HIV-negative patients, the overall response rate was 80% (95% CI: 44, 98), and the mean duration of response was 10.5 months (95% CI: 3.9, 24.2).

The most common (in ≥30% of patients) side effects in patients receiving pomalidomide were decreased absolute neutrophil counts or white blood cells; increased creatinine or glucose; rash; constipation; fatigue; decreased hemoglobin, platelets, phosphate, albumin or calcium; increased ALT; nausea; and diarrhea.

The recommended pomalidomide dose for Kaposi sarcoma is 5 mg orally once daily, with or without food, on days 1 through 21 of each 28-day cycle until disease progression or unacceptable side effects. In patients with AIDS-associated Kaposi sarcoma, highly active antiretroviral therapy should be continued as HIV treatment.