FDA Approves Pomalidomide for Certain Adults With Kaposi Sarcoma
On May 14, 2020, the FDA approved pomalidomide (Pomalyst; Celgene) for the treatment of adults with AIDS-related Kaposi sarcoma that did not respond to highly active antiretroviral therapy and Kaposi sarcoma in adults who are HIV-negative. This drug was granted with priority review and breakthrough therapy designations by the FDA.
This approval was based on efficacy data from the open-label, single-arm Study 12-C-0047 clinical trial, in which 28 patients (18 HIV-positive, 10 HIV-negative) received pomalidomide 5 mg once daily on days 1 through 21 of each 28-day cycle until disease progression or unacceptable toxicity occurred. Of note, all patients who are HIV-positive continued receiving highly active antiretroviral therapy.
The main efficacy end point was overall response rate (ORR), including complete response, clinical complete response, and partial response. Among the 18 HIV-positive patients, the ORR was 67% (95% CI, 41-87) and the median duration of response was 12.5 months (95% CI, 6.5-24.9). Among the 10 HIV-negative patients, these values were 80% (95% CI, 44-98) and 10.5 months (95% CI, 3.9-24.2), respectively.
The most frequently reported (≥30%) adverse reactions tied to pomalidomide use were decreased absolute neutrophil count or white blood cells, elevated creatinine or glucose, rash, constipation, fatigue, decreased hemoglobin, platelets, phosphate, albumin, or calcium, increased alanine aminotransferase, nausea, and diarrhea.—Hina Porcelli
Source: US Food and Drug Administration. FDA grants accelerated approval to pomalidomide for Kaposi sarcoma. Updated May 15, 2020. www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pomalidomide-kaposi-sarcoma. Accessed May 15, 2020.
