HQ Team
August 9, 2024: Citius Pharmaceuticals, Inc., a US-based biotechnology company, got a green light from the country’s drug regulator for its immunotherapy treatment for a rare form of blood cancer that affects the skin.
The Cranford, New Jersey-based firm’s Lymphir drug treats patients with relapsed or refractory cutaneous T-cell lymphoma after at least one prior systemic therapy, according to a company statement.
It is the only cutaneous T-cell lymphoma (CTCL) therapy that targets the interleukin-2 (IL-2) receptor found on malignant T-cells and Tregs. It is expected to be in the US market within the next five months.
The interleukin-2 receptor (IL-2R) is a crucial component of the immune system, specifically involved in the signalling processes of T cells, which are vital for immune responses.
Relapsed, refractory
The CTCl is a cancer that primarily affects the skin but originates from T-lymphocytes, a type of white blood cell involved in the immune response. When CTCL is described as relapsed, it means the cancer has returned after treatment.
If it is refractory, it indicates that the cancer did not respond to treatment at all. Because CTCL arises from T-cells, which are part of the blood and immune system, it is classified as a hematologic malignancy, meaning it is a type of cancer that affects blood cells.
The therapy’s approval by the US Food and Drug Administration is the first since 2018.
The approval includes a postmarketing requirement from the FDA to characterize the risk of visual impairment in CTCL patients treated with Lymphir. The FDA had in June, last year rejected Lymphir and had sought additional product testing data.
No cumulative toxicity
The current FDA clearance was based on end-stage results that demonstrated a 36% objective response rate, reduction in skin disease in 84% of patients, clinically significant pruritis improvement, and no cumulative toxicity.
“Lymphir offers new hope for patients suffering from cutaneous T-cell lymphoma, a rare and chronic cancer characterized by debilitating skin lesions and severe itching,” said stated Leonard Mazur, Chief Executive Officer of Citius Pharmaceuticals.
“This approval is 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 and grow the overall market, currently estimated to be $300-$400 million,” he said.
“We believe Lymphir’s unique IL-2 receptor-targeted treatment, which kills tumour cells directly and concurrently depletes host Tregs to boost the body’s immune response, is an important differentiator and offers clinically meaningful benefits to a significant percentage of r/r patients.”
Non-Hodgin lymphoma
Cutaneous T-cell lymphoma is a rare and often debilitating chronic non-Hodgkin lymphoma that primarily affects the skin.
About 2,500-3,000 patients are diagnosed each year with an estimated 40,000 living with the disease, according to the statement.
Patients with relapsed or refractory CTCL have limited treatment options. They typically cycle through several skin-directed therapies before the cancer becomes resistant or progressive at which point systemic agents are needed to achieve effective disease control.
Once the disease reaches the tumour stage, the cancer is highly malignant and can spread to the lymph nodes and internal organs, resulting in a poor prognosis.
The median overall survival is often less than 12 months in the absence of more aggressive interventions like allogeneic stem cell transplantation. Studies indicate that the median progression-free survival for these patients can be around 1.2 years.
More men than women
CTCL affects men twice as often as women and is typically first diagnosed in patients between the ages of 50 and 60 years of age.
Other than allogeneic stem cell transplantation, for which only a small fraction of patients qualify, there is currently no curative therapy for advanced CTCL.
Reducing and controlling skin plaques and itching without cumulative toxicity is a primary goal of CTCL treatment.
Lymphir offered a mechanism of action designed to target and eradicate malignant T-cells while preserving healthy tissue during the treatment.
“Lymphir’s median time-to-response of only 1.4 months offers many patients rapid skin relief,” added Dr Myron Czuczman, Chief Medical Officer of Citius Pharmaceuticals.
