FDA Approves BMS Drug for Rare Cancer

Rare Daily Staff

The U.S. Food and Drug Administration approved Bristol Myers Squibb’s Pomalyst for patients with AIDS-related Kaposi sarcoma whose disease has become resistant to highly active antiretroviral therapy (HAART), or in patients with Kaposi sarcoma who are HIV-negative. 

Kaposi sarcoma is a rare form of cancer that usually presents as skin lesions but can also develop in several other areas of the body including the lungs, lymph nodes, and digestive system. It mostly affects people who are immunocompromised. Pomalyst, an oral therapy, is the first new treatment option available for those with Kaposi sarcoma in more than 20 years.

Pomalyst is a thalidomide analogue indicated for the treatment of adult patients in combination with dexamethasone, for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or within 60 days of completion of the last therapy.

The approval of Pomalyst was based on the findings of a phase 1/2 open-label, single-arm study evaluating the safety, pharmacokinetics and efficacy of Pomalyst in patients with HIV-positive and HIV-negative symptomatic Kaposi sarcoma, the majority of whom had advanced disease.

Pomalyst was granted accelerated approval, Breakthrough Therapy designation and Orphan Drug designation in these indications based on overall response rates observed in a phase 1/2 open label, single-arm clinical trial. Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.

The primary endpoint of the study was overall response rate, which included complete response, clinical complete response, and partial response, as assessed by investigators according to the AIDS Clinical Trial Group Oncology Committee response criteria for Kaposi sarcoma. For all patients, the ORR was 71 percent with 14 percent of patients achieving complete response and 57 percent a partial response. The median duration of response for all patients was 12.1 months.

“Patients with Kaposi sarcoma have had few options to manage their disease for two decades,” said Diane McDowell, vice president of hematology global medical affairs for Bristol Myers Squibb. “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 drug’s label includes a boxed warning. Pomalyst can cause fetal harm and is contraindicated in females who are pregnant. Pomalyst is only available through a restricted distribution program, Pomalyst REMS. Deep vein thrombosis, pulmonary embolism, myocardial infarction and stroke can occur in patients treated with Pomalyst and thromboprophylaxis is recommended.

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

Photo: Robert Yarchoan, chief of the HIV and AIDS Malignancy Branch within the Center for Cancer Research of the National Cancer Institute