Enterprise Therapeutics, a biotechnology company specializing in respiratory disease therapies, has earned the “rare pediatric disease designation” (RPD) for its investigational cystic fibrosis (CF) treatment, ETD001, from the US Food and Drug Administration (FDA). This designation recognizes the severity and limited treatment options available for CF, a life-threatening genetic disorder primarily affecting children.
ETD001, a novel low-molecular-weight compound, targets the sodium channel (ENaC) in airway epithelium, increasing mucus hydration and clearance. This mechanism aims to alleviate the severe congestion and inflammation caused by failed mucociliary clearance in CF patients’ lungs.
In July 2024, Enterprise commenced a Phase 2a trial to evaluate ETD001’s safety and efficacy in approximately 10% of CF patients with the highest unmet medical needs, either ineligible for or not receiving CFTR modulator therapy.
Preclinical studies have shown that ETD001 has a long-acting effect and has been well-tolerated in healthy subjects during a Phase 1 trial. The FDA’s RPD designation highlights the potential of ETD001 to address the critical unmet medical need for effective CF treatments.
The designation provides Enterprise incentives and support from the FDA during ETD001’s development, including potential access to a Priority Review Voucher (PRV) upon marketing approval. This recognition underscores Enterprise’s commitment to advancing innovative therapies for CF patients with the greatest need.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
