On September 28, 2022 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") ( Nasdaq: CTXR), a late-stage biopharmaceutical company developing and commercializing first-in-class critical care products, reported that the Company has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for denileukin diftitox (I/ONTAK), an engineered IL-2-diphtheria toxin fusion protein for the treatment of patients with persistent or recurrent cutaneous T-cell lymphoma (CTCL) (Press release, Citius Pharmaceuticals, SEP 28, 2022, View Source [SID1234621534]). I/ONTAK is a purified and more bioactive formulation of previously FDA-approved ONTAK. The BLA is supported by a pivotal Phase 3 study (NCT01871727). Results of the study were consistent with the prior FDA-approved formulation.
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"The treatment of advanced cutaneous T-cell lymphoma remains a complex and challenging unmet medical need. Each year, thousands of patients are diagnosed with CTCL, a debilitating orphan disease with no single standard of care. Patients are often treated with multiple alternate therapies. Citius is proud to advance the only potential CTCL therapeutic with a mechanism of action that delivers a cytotoxic protein by binding to the IL-2 receptors found in malignant T-cells and immunosuppressive T-regulatory cells. We look forward to continuing to engage with the FDA as they review our BLA and bringing this treatment option to patients, if approved," stated Leonard Mazur, Chairman and CEO of Citius. "The BLA filing for denileukin diftitox marks the first of our pipeline candidates to be submitted for FDA approval."
About I/ONTAK
I/ONTAK is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. I/ONTAK, a purified version of denileukin diftitox, is a reformulation of previously FDA-approved oncology treatment ONTAK. ONTAK was marketed in the U.S. from 1999 to 2014, when it was voluntarily withdrawn from the market. Manufacturing improvements resulted in a new formulation, which maintains the same amino acid sequence but features improved purity and bioactivity. The new formulation received regulatory approval in Japan in 2021 for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). In 2011 and 2013, the FDA granted orphan drug designation (ODD) to I/ONTAK for the treatment of PTCL and CTCL, respectively.
About Cutaneous T-cell Lymphoma
Cutaneous T-cell lymphoma is a type of cutaneous non-Hodgkin lymphoma (NHL) that comes in a variety of forms and is the most common type of cutaneous lymphoma. In CTCL, T-cells, a type of lymphocyte that plays a role in the immune system, become cancerous and develop into skin lesions, leading to a decrease in the quality of life of patients with this disease due to severe pain and pruritus. Mycosis Fungoides (MF) and Sézary Syndrome (SS) comprise the majority of CTCL cases. Depending on the type of CTCL, the disease may progress slowly and can take anywhere from several years to upwards of ten to potentially reach tumor stage. However, once the disease reaches this stage, the cancer is highly malignant and can spread to the lymph nodes and internal organs, resulting in a poor prognosis. Given the duration of the disease, patients typically cycle through multiple systemic agents to control disease progression. 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. Approximately 3,000 new cases are reported in the United States every year, with an estimated 30,000 – 40,000 individuals living with the disease.