Certa Therapeutics, a biotechnology company developing precision therapies for inflammatory and fibrotic diseases, has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for its lead drug candidate, FT011, for the treatment of Systemic Sclerosis (SSc). This follows the U.S. Food and Drug Administration (FDA) granting both ODD and Fast Track designation for FT011 for the same indication.

FT011 is an innovative oral therapy targeting GPR68, a membrane GPCR receptor that plays a crucial role in fibrosis. The drug has shown promising efficacy in preclinical and early clinical studies, demonstrating its safety, pharmacokinetics, and effectiveness in treating chronic fibrosis in multiple organs.

Systemic Sclerosis is a severe autoimmune disease that causes inflammation and fibrosis in the skin and various internal organs, including the lungs, kidneys, and heart. This fibrosis leads to significant functional impairment and disability. Currently, there are no treatments that can effectively stop or reverse this scarring.

Professor Darren Kelly, CEO and founder of Certa Therapeutics, commented, “Inhibiting GPR68 can alter the biological pathways that cause inflammation and fibrosis in SSc. The EMA's Orphan Drug Designation, in addition to the FDA's designations, highlights the potential of FT011 to meet the needs of SSc patients.”

Certa is preparing for a Phase IIb clinical trial to further evaluate FT011 in SSc. The company is also developing biomarkers and gene signatures to identify patients who are most likely to respond to the treatment, aiming to optimize outcomes.

Orphan Drug Designation from the FDA and EMA is given to products intended to treat rare diseases affecting fewer than 200,000 people in the U.S. or fewer than 5 in 10,000 people in Europe. This designation provides benefits such as potential marketing exclusivity, reduced regulatory fees, and, in Europe, a centralized approval process.

Scleroderma is a debilitating autoimmune disease characterized by inflammation and fibrosis of the skin and internal organs. This condition leads to significant morbidity and a substantial impact on the quality of life, often causing mobility issues, pain, fatigue, and mental health challenges. It has the highest mortality rate among rheumatic diseases.

 

Source: globenewswire.com

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