Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.¹ Scleroderma is an extremely debilitating, potentially life-threatening autoimmune condition characterised by inflammation and fibrosis of the skin and other organs (commonly the lungs, kidneys, and heart).

"We are very pleased that the FDA has granted Orphan Drug Designation to FT011 which we believe highlights the urgent need for innovation and new therapeutic options for scleroderma patients" said Professor Darren Kelly, Certa Therapeutics CEO and founder. "This designation represents an important milestone in the development of FT011, which has the potential to establish first-in-class clinical benefits by precisely targeting the root cause of fibrosis and offer treatment across multiple organs within these patients."

Certa previously announced positive top-line results from a Phase 2 study which indicated that treatment with FT011 for 12 weeks resulted in a clinically meaningful improvement in 60% of patients treated with FT011 400mg (p = 0.019 vs. placebo) and 20% of patients in the FT011 200mg group compared with 10% in the placebo group.² Three patients in the pooled FT011 groups achieved a maximum  CRISS score of 1.0, representing the greatest probability of clinical improvement.³ The study safety profile demonstrated that FT011 was safe and well tolerated, with no differences in adverse event (AE) rates between the treatment arms. There were no serious AEs reported in the study, nor any AEs resulting in study drug interruption, withdrawal, or discontinuation.

Data from the Phase 2 study will be presented at the American College of Rheumatology Annual Meeting 2023 (ACR Convergence), taking place between 10-15 November 2023, in San Diego CA.