Certa Therapeutics Gets EU Orphan Status for FT011 in Treating Systemic Sclerosis

On July 22, 2024, Certa Therapeutics (Certa), a biotechnology company specializing in precision therapies for inflammatory and fibrotic diseases, announced a significant milestone. The European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to Certa's leading drug candidate, FT011, for the treatment of Systemic Sclerosis (SSc). This follows previous designations of ODD and Fast Track status for FT011 by the U.S. Food and Drug Administration (FDA) for the same condition.

FT011 is a groundbreaking, first-in-class oral therapy designed to treat chronic fibrosis across multiple organs. Its mechanism of action involves targeting GPR68, a critical but previously untapped membrane GPCR receptor that acts as a master regulator of fibrosis. Comprehensive data has shown promising results in various in vitro and in vivo models of inflammatory and fibrotic diseases. Clinical studies, specifically Phase I and IIa trials involving SSc patients, have demonstrated its favorable efficacy, safety, and pharmacokinetic profiles.

Systemic Sclerosis is a chronic and progressive autoimmune condition characterized by inflammation and fibrosis—also known as scar tissue formation—in the skin and several internal organs, including the lungs, kidneys, and heart. The condition is marked by skin fibrosis, which significantly impairs function and causes disability in patients. Currently, no treatments effectively halt or reverse the scarring associated with SSc.

Professor Darren Kelly, CEO and founder of Certa Therapeutics, highlighted the importance of GPR68 inhibition in modulating the biological pathways that lead to inflammation and fibrosis in SSc patients. He noted that the designations by both the EMA and FDA underscore the potential of FT011 to meet a critical need for those suffering from this debilitating condition. These regulatory recognitions validate Certa's efforts and emphasize the need for innovative treatments to address chronic fibrotic diseases, which often have poor prognoses.

Certa is planning a Phase IIb confirmatory clinical trial for FT011 in SSc patients, aiming to provide better treatment options for fibrosis. Additionally, the company is working on developing biomarkers and gene signatures to identify patients who are most likely to respond to the treatment, ensuring the best possible outcomes.

The FDA and EMA grant orphan status to products aimed at treating, diagnosing, or preventing life-threatening rare diseases affecting fewer than five in 10,000 people in Europe or under 200,000 people in the U.S. These designations provide various benefits, including potential marketing exclusivity, reduced regulatory fees, and, in the EU, a centralized approval process.

Scleroderma, also known as systemic sclerosis, is a severe and potentially life-threatening autoimmune condition. It involves inflammation and fibrosis of the skin and other organs, such as the lungs, kidneys, and heart. The disease significantly impacts patients' lives, often leading to reduced mobility, pain, fatigue, and severe effects on mental health. It has the highest mortality rate among rheumatic diseases.

Certa Therapeutics is a clinical-stage biotechnology company dedicated to improving patients' lives by developing novel targeted therapies for debilitating diseases. The company has developed a platform of drug candidates and validated the role of GPR68, a specific G protein-coupled receptor (GPCR) involved in inflammatory and fibrotic signaling pathways. GPR68 remains inactive in healthy tissue but is activated following injury or disease. Research has shown that GPR68 affects multiple downstream pathways that cause inflammation and fibrosis.

Certa's innovative drug candidates have shown proof of concept as potential treatments for various serious and chronic fibrotic conditions affecting the kidney, lung, eye, skin, and heart. The impact of fibrotic diseases is significant, contributing to 45% of global deaths, highlighting the urgent need for breakthroughs in this field. The market for such treatments is valued at over $15 billion annually. Certa aims to combine its novel therapeutics with biomarkers and genetic analysis to identify patients most likely to benefit, offering the potential for precision therapy.

FT011 is an investigational product and has not yet received marketing authorization from any regulatory agency. It is currently undergoing clinical trials to assess its safety and effectiveness in treating systemic sclerosis.