Rare Daily Staff

The U.S. Food and Drug Administration (FDA) has granted Celgene a Breakthrough Therapy designation for Pomalyst (pomalidomide) for the treatment of patients with HIV-positive Kaposi sarcoma who have previously received systemic chemotherapy, as well as patients with HIV‐negative Kaposi sarcoma.

Kaposi sarcoma is a multicentric tumor caused by Kaposi sarcoma-associated herpesvirus. It is most common in persons infected with HIV. Patients suffer multiple lesions on the skin and oral mucosa, and at times other organs such as the lungs or gastrointestinal mucosa. Currently, there are no approved therapies for HIV-positive patients who are refractory to or intolerant of systemic chemotherapy.

Although the use of combination anti-retroviral treatments (cART or HAART) has reduced the incidence of advanced Kaposi sarcoma in the United States, there are still nearly 2,000 new cases each year. The disease is more highly prevalent in areas of the world where HIV treatments are less available, such as sub-Saharan Africa, and in some countries is the most common tumor in men overall.

The Breakthrough Therapy designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases. It was granted by the FDA on the basis of the results of a clinical study performed under a Cooperative Research and Development Agreement by a team led by Robert Yarchoan of the HIV and AIDS Malignancy Branch within the Center for Cancer Research of the National Cancer Institutes (NCI). The results of that study evaluated Pomalyst in patients with Kaposi sarcoma, with or without HIV infection, many of whom had received prior cytotoxic chemotherapy.

Celgene also has two additional studies planned in this disease. In partnership with the AIDS Malignancy Consortium, a U.S. multicenter study will be performed to confirm and extend the results of the NCI study. The consortium is also sponsoring a second study in sub-Saharan Africa, where Kaposi sarcoma continues to be a serious problem.

Pomalyst is a thalidomide analogue that is approved, in combination with dexamethasone, to treat patients with multiple myeloma who have received at least two prior therapies, and have demonstrated disease progression within 60 days of completion of the last therapy. 

Celgene plans to submit a supplemental New Drug Application for Pomalyst as a treatment for Kaposi sarcoma by the end of 2019.

“The encouraging news of the FDA Breakthrough Therapy designation for Pomalyst in Kaposi sarcoma reflects the urgency in accelerating the development of therapies to address diseases of this type,” said Jay Backstrom, chief medical officer of Celgene. “We will continue to work closely with the agency to move this program forward for patients with this rare and serious cancer.”

Photo: said Jay Backstrom, chief medical officer of Celgene.

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