The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TCP-25, a novel immunomodulatory peptide developed by Swedish biotech company Xinnate AB for the treatment of Epidermolysis Bullosa (EB), a rare and devastating skin disease.
"We are excited about FDA's decision to grant ODD for TCP-25, recognizing its unique mechanism of action, the robust data generated during preclinical and clinical development and the significant unmet medical needs of patients suffering from EB," said Helene Hartman, CEO of Xinnate. "This designation underscores the potential of TCP-25 as a transformative treatment option and will facilitate our efforts to bring this therapy to patients who needs it most."
Understanding TCP-25's Dual-Action Mechanism
TCP-25 represents a novel approach to treating EB, a condition characterized by extremely fragile skin that blisters and tears from minor friction or trauma. The peptide-based therapy works through a dual-action mechanism, simultaneously targeting inflammation and bacterial infection in wounds—two critical factors that impair healing in EB patients.
This innovative approach could potentially address a significant gap in the current treatment landscape for EB, which primarily focuses on wound care and pain management rather than addressing the underlying pathophysiological mechanisms of impaired healing.
Clinical Development Progress
Xinnate has already completed a three-part Phase 1 study of TCP-25 gel, which included a cohort of patients with Epidermolysis Bullosa. Based on these promising results, the company is planning to initiate Phase 2 clinical trials in 2025 to further evaluate the efficacy and safety of the treatment.
The completed Phase 1 studies have generated robust data supporting TCP-25's potential as a treatment option for EB, demonstrating both safety and preliminary efficacy signals that warrant further investigation.
Significance of Orphan Drug Designation
The FDA grants Orphan Drug Designations to support the development of therapies for rare disorders affecting fewer than 200,000 people in the United States. For Xinnate, this designation provides several important benefits, including:
- Tax credits for qualified clinical trials
- Exemptions from certain FDA application fees
- Seven years of market exclusivity following drug approval
These incentives are designed to accelerate the development of treatments for rare diseases like EB, where the limited patient population might otherwise make drug development economically challenging.
Epidermolysis Bullosa: A Rare Disease with Significant Unmet Needs
Epidermolysis Bullosa is a group of rare genetic disorders that affect the connective tissue and cause severe skin fragility. Patients with EB develop painful blisters and wounds from the slightest friction or trauma, leading to significant morbidity and, in severe cases, premature mortality.
The condition has profound impacts on quality of life, with patients requiring intensive wound care and pain management. Current treatment options are limited primarily to supportive care, highlighting the urgent need for therapies that can address the underlying disease mechanisms.
About Xinnate AB
Xinnate is a clinical-stage pharmaceutical company based in Lund, Sweden, focused on developing novel peptide-based therapies that target the interplay between microbial presence and inflammatory responses in wound healing. The company is a member of SmiLe Venture Hub at Medicon Village.
With TCP-25 as its lead candidate, Xinnate aims to transform the treatment landscape for patients with EB and potentially expand to other inflammatory skin conditions where dysfunctional healing represents a significant clinical challenge.
