The US Food and Drug Administration (FDA) has approved Citius Pharmaceuticals’ Lymphir (denileukin diftitox-cxdl) to treat certain cases of cutaneous T-cell lymphoma (CTCL).
The immunotherapy has been specifically authorised for use in adults with relapsed or refractory disease who have received at least one prior systemic therapy.
Approximately 40,000 people in the US are living with CTCL, a rare type of non-Hodgkin lymphoma that primarily affects the skin and causes red, itchy, scaly patches.
Patients usually receive several skin-directed therapies before their cancer becomes resistant or progressive, at which point systemic agents are required to achieve effective disease control.
Lymphir is now the only CTCL therapy that targets the interleukin-2 (IL-2) receptor found on malignant T-cells and Tregs, and is the first FDA-approved product for Citius.
The US regulator’s decision was supported by positive results from a late-stage study, in which Lymphir demonstrated an objective response rate of 36.2% and a complete response rate of 8.7%.
The median time to response was 1.41 months and approximately 70% of responders saw results after one to two cycles of treatment.
The drug was also shown to reduce skin disease in 84.4% of patients, with 12.5% achieving complete skin disease clearance. Clinically significant improvements in itch, which was evaluated as an exploratory endpoint, were also demonstrated.
“We believe Lymphir’s unique IL-2 receptor-targeted treatment, which kills tumour 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,” said Myron Czuczman, chief medical officer at Citius.
The company’s chief executive officer, Leonard Mazur, described the approval as a “significant milestone” for CTCL patients.
“The introduction of Lymphir, with its potential to rapidly reduce skin disease and control symptomatic itching without cumulative toxicity, is expected to expand the CTCL treatment landscape,” he said.
Mazur added that the company is preparing to launch Lymphir in the US market “within the next five months” and is “dedicated to working closely with healthcare providers” to ensure that all eligible patients have timely access to the therapy.