Pharmaceutical Business review

Xentria announces FDA orphan drug designation for XTMAB-16 in sarcoidosis

The US FDA’s Center for Drug Evaluation and Research. (Credit: The U.S. Food and Drug Administration)

TNF-α is a proinflammatory cytokine that has been implicated in the pathogenesis of sarcoidosis and associated granuloma formation.

“We are pleased the FDA has granted Orphan Drug Designation for XTMAB-16 for the treatment of sarcoidosis, as it reaffirms the need for an effective therapy for patients with this potentially life-threatening inflammatory disease,” said Thomas Shea, President of Xentria. “This is an important regulatory milestone for our company, and we look forward to advancing XTMAB-16 into the clinical phase.”

Founded in April 2020, Xentria is an innovative biopharmaceutical company with the primary focus of building an expansive product pipeline of cost-effective and efficacious biologics and biosimilars to treat rare diseases, immunologic, metabolic, musculoskeletal disorders, and certain types of cancer.

The FDA Office of Orphan Products Development grants orphan status to drugs which are being developed to treat rare disorders affecting fewer than 200,000 people in the U.S., addressing unmet medical needs for these patients. This designation provides certain benefits to the drug developer, including seven years of market exclusivity upon FDA approval, prescription drug user fee waivers and tax credits for qualified clinical trials. 1 2

Sarcoidosis is a chronic, multisystem inflammatory disorder of unknown etiology that is characterized by the presence of noncaseating epithelioid granulomas, accompanied by infiltration of mononuclear cells and destruction of microarchitecture. The disease can affect the skin, eyes, heart, and central nervous system, and >90% of cases involve the lungs.

Symptoms range from asymptomatic to severe—including respiratory insufficiency, blindness, neurological disease, and cardiac death. While medications for systemic organ involvement often control this disease, some patients fail to respond to initial treatments and require additional targeted therapy, resulting in significant costs and treatment burdens. This disease presents a significant unmet medical need and a very important area of research and development for Xentria.

XTMAB-16 is a chimeric human-murine IgG1κ anti-TNFα monoclonal antibody with a molecular weight of ~149 kDa being developed as a novel biologic product for the treatment of patients with sarcoidosis with or without extrapulmonary involvement.

No TNF-α inhibitor is currently approved for the treatment of sarcoidosis. Extensive analyses have been conducted to demonstrate the physio-chemical properties and pharmacology of XTMAB-16 as a TNF-α inhibitor.

Xentria, whose name stems from the word “centrality,” was founded in 2020 and is an innovative biopharmaceutics company whose primary focus is to build an expansive product pipeline of cost-effective and efficacious biologics and biosimilars to address critical unmet need for patients suffering from rare diseases, immunologic, metabolic, and musculoskeletal disorders, as well as oncology.

Headquartered in Chicago, with partners in Israel and China, the company is dedicated to research and development, clinical science, manufacturing, and commercialization and driven to treat the clinical and social burdens of diseases while fostering sustainable growth.

Source: Company Press Release