FDA Grants Accelerated Approval to Pomalidomide for Kaposi Sarcoma
On May 14, 2020, the U.S. Food and Drug Administration (FDA) expanded the indication (https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pomalidomide-kaposi-sarcoma) of pomalidomide (Pomalyst®) to include treating adult patients with AIDS-related Kaposi sarcoma after failure of highly active antiretroviral therapy and Kaposi sarcoma in adult patients who are HIV negative.
Efficacy was investigated in a National Cancer Institute-funded, open-label, single-arm clinical trial (Study 12-C-0047) in which 28 patients (18 HIV positive and 10 HIV negative) received 5 mg of pomalidomide orally once daily on days 1–21 of each 28-day cycle until disease progression or unacceptable toxicity. All HIV-positive patients continued highly active antiretroviral therapy.
The main efficacy outcome measure was overall response rate (ORR), which included complete response, clinical complete response, and partial response. The investigator assessed the response according to the AIDS Clinical Trial Group Oncology Committee response criteria for Kaposi sarcoma. Among the 18 HIV-positive patients, ORR was 67% (95% CI = 41, 87) with a median response duration of 12.5 months (95% CI = 6.5, 24.9). Among the 10 HIV-negative patients, ORR was 80% (95% CI = 44, 98) with a median response duration of 10.5 months (95% CI =3.9, 24.2).
The most common adverse reactions (≥ 30%), including laboratory abnormalities, were decreased absolute neutrophil count or white blood cells, elevated 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 taken orally once daily with or without food on days 1–21 of each 28‑day cycle until disease progression or unacceptable toxicity. Continue highly active antiretroviral therapy as HIV treatment in patients with AIDS-related Kaposi sarcoma.
FDA approved the indication under accelerated approval based on ORR. Continued approval for the indication may be contingent on verification and description of clinical benefit in confirmatory trials.
FDA granted pomalidomide priority review designation and breakthrough therapy designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions—Drugs and Biologics (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics).
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System (https://www.accessdata.fda.gov/scripts/medwatch/index.cfm) or by calling 800-FDA-1088.
For assistance with single-patient oncology investigational new drug applications, contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov (mailto:OncProjectFacilitate@fda.hhs.gov).