Enterprise Therapeutics has granted a rare paediatric disease designation (RPD) from the FDA for its cystic fibrosis investigational therapy.
ETD001 is a small molecule therapeutic that targets the ENaC sodium channel to enhance airway hydration and mucous clearance.
The company dosed the first person with cystic fibrosis in its Phase IIa trial of ETD001 back in July 2024.
ETD001's safety and efficacy profile is currently being assessed in the 10% of cystic fibrosis patients with the highest unmet medical need.
The RPD designation was based on the assessment of CF as a serious or life-threatening disease, primarily affecting individuals aged from birth to 18 years.
Cystic fibrosis is estimated to affect more than 100,000 globally, with the average life expectancy of somebody with the disease being only 50 years.
Failing to clear mucous from the lungs leads to cycles of infection and inflammation, which results in an ongoing decline in lung function.
ETD001 aims to increase the volume of fluid in the lungs, which can hydrate the problem mucous and improve clearance — resulting in substantial improvements in lung function.
CEO of Enterprise Therapeutics, Dr John Ford said: “We’re delighted with the FDA’s decision and would like to thank the Office of Pediatric Therapeutics and the Office of Orphan Products Development for their consideration. As we progress though our Phase IIa trial of ETD001, this RPD designation will further support our mission to advance this novel approach for treating pwCF with the highest unmet medical need, as rapidly as possible.”
Annabella Amatulli, Head of Regulatory Affairs, added: “We are thrilled to be granted the RPD designation by the FDA, a regulatory framework intended to encourage and accelerate innovative therapies, in recognition of the significance of our programme in addressing an unmet medical need."
"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 Priority Review Voucher (PRV) at the time of marketing approval.”