FDA Grants Orphan Drug Status to EydisBio's TAK1 Inhibitor for Systemic Sclerosis

EydisBio, Inc., based in Durham, North Carolina, announced on September 25, 2024, that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to EYD-001, a highly selective and potent TAK1 inhibitor designed for the treatment of systemic sclerosis. This recognition offers hope for a novel therapeutic approach to a debilitating autoimmune disease.

Systemic sclerosis is a rare and severe autoimmune disorder characterized by the hardening and fibrosis of the skin and internal organs, resulting in substantial morbidity and mortality. Central to its development is the transforming growth factor-β (TGF-β) pathway, with TAK1 being a critical component. Activation of this pathway leads to inflammation and fibrosis, which are primary features of systemic sclerosis. Inhibiting this pathway through TAK1 offers a promising therapeutic strategy.

EydisBio, an early-stage pharmaceutical company, is leveraging innovative approaches to treat various autoimmune and inflammatory diseases, including systemic sclerosis. Last year, the company, in collaboration with Dr. John Varga and his ScleroLab research group at the University of Michigan, published preclinical data. These studies demonstrated that EYD-001, previously known as HS-276, significantly reduced dermal thickening and p-TAK1 expression in a mouse model of systemic sclerosis induced by bleomycin. Additionally, in skin fibroblasts derived from patients, treatment with EYD-001 markedly decreased the mRNA expression of fibroinflammatory genes and blocked TGFβ-mediated increases in fibrotic protein expression.

The FDA's Orphan Drug Designation underscores the potential of EYD-001 to address a significant unmet medical need. This designation is aimed at drugs intended to treat rare diseases affecting fewer than 200,000 people in the United States. It offers several benefits, including seven years of market exclusivity upon approval, tax credits for clinical trial costs, and support in clinical trial design. Additionally, it waives prescription drug user fees, making the drug development process more feasible and less financially burdensome for companies.

Dr. Tim Haystead, Founder and President of EydisBio, expressed excitement over the FDA's recognition. He emphasized the potential of EYD-001 to significantly impact the lives of systemic sclerosis patients and highlighted the innovative nature of the company's research. Dr. Haystead also noted that this designation strengthens EydisBio's commitment to bringing EYD-001 to patients as efficiently and safely as possible.

EydisBio is dedicated to advancing its TAK1 inhibitor program and intends to initiate clinical trials shortly. The company will continue to collaborate closely with the FDA to ensure that this promising treatment reaches patients suffering from systemic sclerosis promptly.

The Orphan Drug Designation is a pivotal milestone for EydisBio, reflecting the company's progress and dedication to developing treatments for rare diseases. The incentives associated with this designation will enable EydisBio to advance its clinical programs more effectively, ultimately benefiting patients who have limited treatment options.