FDA Awards Rare Pediatric Disease Status to Enterprise's Cystic Fibrosis Drug

The US Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation to Enterprise Therapeutics’ experimental treatment for cystic fibrosis. Affecting over 100,000 people worldwide, cystic fibrosis is a genetic disorder that leads to the accumulation of thick mucus in the lungs, digestive system, and other organs. This condition typically begins in early childhood, with symptoms varying between individuals but generally becoming more severe over time, causing extensive damage to the affected organs.

Enterprise Therapeutics has developed ETD001, a therapy targeting the sodium channel in the airway epithelium. This mechanism aims to enhance mucus hydration and clearance. In a phase 1 trial involving healthy participants, ETD001 exhibited a well-tolerated profile and demonstrated long-acting properties in pre-clinical studies. Currently, a phase 2a trial is underway to evaluate the safety and efficacy of ETD001 in cystic fibrosis patients who are either ineligible for or not receiving cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy.

The FDA’s RPD designation is intended to promote the development of drugs for serious or life-threatening diseases affecting fewer than 200,000 individuals in the US, primarily targeting patients under the age of 18. As part of this program, sponsors whose products receive approval can qualify for a priority review voucher (PRV). This voucher can be used to expedite the review process of another drug or can be sold or transferred to another sponsor.

John Ford, the CEO of Enterprise Therapeutics, expressed optimism about the RPD designation, stating: “As we progress through our phase 2a trial of ETD001, this RPD designation will further support our mission to advance this novel approach for treating people with cystic fibrosis with the highest unmet medical need, as rapidly as possible.”

Annabella Amatulli, head of regulatory affairs at Enterprise, emphasized the benefits of the RPD designation, adding: “The RPD designation will give Enterprise access to valuable incentives and support from the FDA during the development of ETD001, including the eligibility to request a PRV at the time of marketing approval.”

The FDA’s decision follows closely on the heels of Enterprise’s announcement that the first cystic fibrosis patient had been dosed in its phase 2a trial. Ford described this development as “an incredible milestone,” highlighting the significance of the progress being made in the treatment of this challenging condition.