FDA Approves New Immunotherapy for Rare Blood Cancer
A new immunotherapy option is available for adults with advanced cutaneous T-cell lymphoma, a rare and challenging form of blood cancer. The U.S. Food and Drug Administration approved denileukin diftitox (Lymphir) this week, the first therapy approved for this disease since 2018.
Cutaneous T-cell lymphoma is a rare type of non-Hodgkin lymphoma that primarily affects the skin, causing painful lesions and severe itching. While the disease often progresses slowly, it can become aggressive and spread to lymph nodes and internal organs, leading to a poor prognosis. With approximately 3,000 new cases diagnosed each year in the U.S., treatment options have been limited, leaving patients to cycle through multiple therapies as the disease becomes resistant.
Denileukin diftitox is the first and only systemic treatment that targets the interleukin-2 receptor on malignant T cells and regulatory T cells, offering a new approach to managing this difficult-to-treat cancer. Its approval was supported by results from a phase 3 study that evaluated 112 patients with advanced disease who had already undergone at least one prior therapy. Moffitt Cancer Center was a participating site for this trial.
Yumeng Zhang, MD
Findings showed 36% of patients achieved an objective response, meaning they experienced a significant reduction in cancer symptoms, with 9% achieving a complete response, where all signs of cancer disappeared. Additionally, 84% of patients showed a reduction in skin disease, and 52% of those who responded maintained their response for at least six months. Notably, denileukin diftitox provided rapid relief, with a median time of response of just over one month.
“Lymphir is the first therapeutic option in many years to offer hope of reducing skin disease, bringing us one step closer to filling the need for cutaneous T-cell lymphoma patients, particularly those who are unable to complete or continue prior therapies,” said Yumeng Zhang, MD, a medical oncologist in Moffitt’s Malignant Hematology Department.
