FDA Approves Lymphir for Relapsed, Refractory Cutaneous T-Cell Lymphoma

The FDA has approved Lymphir, an immunotherapy drug to help treat patients with cutaneous T-cell lymphoma who received at least one previous therapy.

The Food and Drug Administration has approved Lymphir (denileukin diftitox-cxdl) for patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL) who have received at least one previous treatment.

Lymphir is a drug that targets the interleukin-2 (IL-2) receptor that is found on cancerous T-cells and regulatory T-cells (Tregs), according to a news release from Citius Pharmaceuticals, the manufacturer of Lymphir. Tregs suppress anti-tumor immune response, meaning it makes it difficult for the body to destroy the cancer cells. However, they are used as targets for immunotherapy drugs, according to a study in Nature.

“We believe Lymphir’s unique IL-2 receptor-targeted treatment, which kills tumor cells directly, and concurrently depletes host Tregs in order to boost the body's immune response, is an important differentiator and offers clinically meaningful benefits to a significant percentage of [relapsed or refractory] patients,” Dr. Myron Czuczman, chief medical officer of Citius Pharmaceuticals, said in the news release. “As the only IL-2 receptor-targeted immunotherapy for CTCL, Lymphir provides a novel and non-cross-resistant treatment option without cumulative toxicity for [patients with] stage 1 to 3 relapsed or refractory [CTCL] for whom symptomatic skin involvement interferes with their daily quality of life. Lymphir’s median time-to-response of only 1.4 months offers many patients rapid skin relief.”

The approval of Lymphir is based on Study 302, a phase 3 trial that included approximately 119 patients, according to the release. The primary efficacy population included 69 patients with stage 1 to 3 CTCL and were treated with Lymphir. Researchers reported that the objective response rate (percentage of patients whose tumors shrunk or disappeared after treatment) was 36.2%. Of note, 8.7% of patients achieved a complete response, meaning their disease disappeared after treatment, the release stated.

The median time to response to the treatment was 1.41 months, and approximately 70% of patients experienced results after one to two cycles of treatment, the release noted. The duration of response in 52% of patients was at least 6 months. For patients who were skin-evaluable, 84.4% had a decrease in skin tumor burden and 12.5% experienced complete clearance of skin disease.

Skin itching (pruritus) was evaluated as an exploratory endpoint (main result measured at the end of a study to see if treatment worked). Researchers found that 31.7% of patients demonstrated a clinically significant improvement in skin itching.

Common side effects occurring in at least 20% of patients included increased transaminases (liver stress), albumin decreased (problems with the liver or kidneys), edema (swelling from trapped fluid in body tissue), nausea, fatigue, rash, chills, constipation, hemoglobin decreased, musculoskeletal pain, fever and capillary leak syndrome (blood escaping into capillary walls).

"As a treating oncologist, I have seen the profound negative effect on the quality of life in patients with relapsed or refractory CTCL. Given the long-term nature of the disease, pruritus, ulceration of the tumors and secondary pyogenic skin infection, it is vital to get this skin involvement under control,” Dr. Francine Foss, professor of hematology and Director of the Multidisciplinary T-cell Lymphoma Program at Yale Cancer Center, said in the release. “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 CTCL patients, particularly those that are not able to complete or continue prior therapies.”

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