NEW YORK: The US Food and Drug Administration (FDA) has granted approval for Lymphir, a new therapy developed by Citius Pharmaceuticals, to treat a specific type of rare blood cancer, marking a significant milestone for patients who have exhausted other treatment options.
The approval comes after the FDA previously rejected the therapy a year ago, requesting additional product testing data.
Lymphir is now authorized for use in adult patients with relapsed cutaneous T-cell lymphoma (CTCL), a rare form of blood cancer affecting the skin. This condition is diagnosed in approximately 2,500 to 3,000 persons annually in the US, according to Citius Pharmaceuticals.
The company plans to launch Lymphir within the next five months, pricing it at levels comparable to existing treatments on the market. This decision follows a rigorous review process after the initial rejection in July of the previous year.
Lymphir, also known as denileukin diftitox-cxdl, is a targeted immune therapy designed to address Stage I-III disease after at least one prior systemic therapy. It combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments, targeting IL-2 receptors on the surface of cancer cells. This targeted approach inhibits protein synthesis within the cells, leading to their death.
The therapy has shown promise in depleting immunosuppressive regulatory T lymphocytes (Tregs) and demonstrating antitumor activity through direct cytocidal action on IL-2 receptor-expressing tumors.
However, the FDA has issued a serious “boxed warning” for Lymphir, highlighting the risk of capillary leak syndrome, a potentially life-threatening condition where blood plasma escapes through tiny blood vessels, leading to a rapid drop in blood pressure.
Lymphir had previously received regulatory approval in Japan for the treatment of CTCL and peripheral T-cell lymphoma (PTCL) in 2021. Subsequently, Citius Pharmaceuticals acquired exclusive rights to develop and commercialize the therapy in markets outside Japan and certain parts of Asia.
