The European Medicines Agency Committee for Orphan Medicinal Products has issued a positive recommendation for orphan drug designation of dusquetide, the active ingredient in SGX945, for the treatment of Behçet Disease. This recommendation follows review of recently published Phase 2a data demonstrating biological efficacy and safety. The recommendation now advances to the European Commission for ratification.
Orphan designation in the European Union provides 10 years of market exclusivity upon approval, along with development incentives and centralized authorization access. This regulatory pathway is designed to encourage development of treatments for rare diseases where patient populations are small and development costs can be prohibitive. SGX945 has previously received orphan drug and fast track designations from the U.S. Food and Drug Administration for Behçet Disease.
Behçet Disease is a rare, chronic inflammatory disorder that affects multiple systems in the body, including blood vessels, skin, mucous membranes, joints, eyes, and the digestive and nervous systems. The condition is characterized by recurrent oral and genital ulcers, skin lesions, and inflammation of the eyes. Current treatment options are limited and primarily focus on managing symptoms rather than addressing the underlying disease mechanisms.
The positive EMA opinion represents a significant regulatory milestone for Soligenix and for patients with Behçet Disease who currently have few treatment options. Orphan drug designation can accelerate the development and approval process for treatments targeting rare diseases, potentially bringing new therapies to market more quickly. The 10-year market exclusivity period provides commercial protection that can help justify the investment required to develop treatments for small patient populations.
Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. The company's Specialized BioTherapeutics business segment is developing dusquetide as part of its first-in-class innate defense regulator technology platform. Additional information about the company's development programs is available in their newsroom at https://ibn.fm/SNGX.
The advancement of dusquetide through the regulatory process highlights ongoing progress in addressing rare diseases that have historically received limited research attention. For patients with Behçet Disease, this development offers hope for a targeted treatment that could potentially modify the course of the disease rather than simply managing symptoms. The regulatory incentives provided through orphan drug designation programs in both Europe and the United States continue to play a crucial role in encouraging pharmaceutical companies to invest in rare disease research.
