The US Food and Drug Administration (FDA) has granted rare paediatric disease (RPD) designation to Enterprise Therapeutics’ investigational cystic fibrosis therapy.
Estimated to affect more than 100,000 people globally, cystic fibrosis is a genetic disease that causes sticky mucus to build up in the lungs, digestive system and other organs in the body.
Symptoms usually begin in early childhood and vary between patients, but the condition gets progressively worse over time and causes increasing damage to the affected organs.
Enterprise’s ETD001, which targets the sodium channel in the airway epithelium to increase the hydration and clearance of mucus, has already demonstrated a well-tolerated profile in a phase 1 trial of healthy participants and has been shown to be long-acting in pre-clinical studies.
A phase 2a trial is now aiming to assess the safety and efficacy of the candidate in patients who are ineligible for, or are not receiving, cystic fibrosis transmembrane conductance regulator modulator therapy.
FDA RPD designation aims to incentivise drug development for serious or life-threatening diseases that affect fewer than 200,000 people in the US and primarily impact patients aged under 18 years.
Under the programme, a sponsor that receives an approval for a RPD product may qualify for a priority review voucher (PRV) that can be redeemed to expedite the review of a different product, or be sold or transferred to another sponsor.
John Ford, Enterprise’s chief executive officer, said: “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 the company, added: “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 comes just two months after Enterprise announced that the first cystic fibrosis patient had been dosed in its phase 2a trial, which Ford said represented “an incredible milestone”.
