Enterprise Therapeutics Granted Rare Pediatric Disease Designation in US for Cystic Fibrosis Therapy ETD001

Enterprise Therapeutics Ltd, a biopharmaceutical firm focused on developing innovative treatments for respiratory diseases, has announced that its investigational cystic fibrosis (CF) therapy, ETD001, has received the 'rare pediatric disease designation' (RPD) from the US Food and Drug Administration (FDA).

ETD001 is a pioneering low molecular weight compound designed to inhibit the sodium channel (ENaC) in the airway lining, enhancing mucus hydration and clearance. On July 23, 2024, Enterprise commenced dosing the first cystic fibrosis patient in its Phase 2a trial of ETD001. This trial aims to establish clinical proof-of-concept by evaluating the safety and effectiveness of ETD001 in around 10% of CF patients who exhibit the most significant unmet medical needs. Specifically, the study will examine lung function (FEV1) in CF patients who are either ineligible for or not receiving CFTR modulator therapy.

The FDA's RPD designation is based on the classification of CF as a severe or life-threatening condition, predominantly impacting individuals from birth to 18 years of age. CF affects more than 100,000 individuals globally, with an average life expectancy of approximately 50 years. The disease is characterized by impaired mucociliary clearance and mucus buildup in the lungs, leading to recurrent infections, inflammation, and progressive lung function decline. By inhibiting ENaC, ETD001 aims to increase lung fluid volume, thus hydrating mucus, enhancing its clearance, reducing congestion, and significantly improving lung function. Previously, ETD001 demonstrated a well-tolerated profile in healthy subjects during a Phase 1 trial and exhibited long-acting properties in pre-clinical studies.

Dr. John Ford, CEO of Enterprise Therapeutics, expressed his enthusiasm about the FDA’s decision. He extended gratitude to the Office of Pediatric Therapeutics and the Office of Orphan Products Development for their consideration. He emphasized that the RPD designation would support the company’s mission to fast-track this novel treatment for CF patients with high unmet medical needs as they move forward with the Phase 2a trial.

Annabella Amatulli, Head of Regulatory Affairs at Enterprise Therapeutics, highlighted the significance of receiving the RPD designation from the FDA. She acknowledged that this regulatory framework is designed to incentivize and expedite the development of innovative therapies that address unmet medical needs. The RPD designation provides Enterprise access to crucial incentives and support from the FDA throughout the development of ETD001, including the possibility of requesting a Priority Review Voucher (PRV) upon marketing approval.

In summary, Enterprise Therapeutics Ltd is making significant strides in the development of ETD001, a novel therapy for cystic fibrosis. The recent RPD designation by the FDA underscores the potential impact of this treatment in meeting the needs of CF patients, particularly those with the greatest unmet medical needs. The ongoing Phase 2a trial will further evaluate the efficacy and safety of ETD001, bringing hope to the CF community for a new and effective treatment option.