ETD001 aims to improve lung function by targeting mucus clearance in patients with high unmet medical needs.
RT’s Three Key Takeaways:
- FDA Designation: The investigational cystic fibrosis therapy ETD001 has been granted rare pediatric disease designation by the FDA, acknowledging its potential to address serious unmet medical needs in children.
- Mechanism of Action: ETD001 targets the sodium channel (ENaC) in the airway epithelium to increase mucus hydration and clearance, with the aim of improving lung function in cystic fibrosis patients.
- Ongoing Clinical Trial: ETD001 is currently being tested in a phase 2a trial, assessing its safety and efficacy in cystic fibrosis patients who are not eligible for or are not receiving CFTR modulator therapy.
Enterprise Therapeutics Ltd, a biopharmaceutical company focused on developing treatments for respiratory diseases, announced its novel cystic fibrosis investigational therapy, ETD001, has been granted rare pediatric disease designation by the US Food and Drug Administration (FDA).
ETD001 is a low molecular weight compound with first-in-class potential, which targets the sodium channel (ENaC) in the airway epithelium to increase the hydration and clearance of mucus.
Enterprise announced the dosing of the first person with cystic fibrosis in its phase 2a trial of ETD001 in July. The trial aims to deliver clinical proof-of-concept and to assess the safety and efficacy of ETD001 in the ~10% of those with cystic fibrosis with the highest unmet medical need. The study will assess lung function in people with cystic fibrosis who are either ineligible for or are not receiving CFTR modulator therapy.
The rare pediatric disease designation was based on the assessment of cystic fibrosis as a serious or life-threatening disease, primarily affecting individuals aged from birth to 18 years.
Potential of ETD001 for Cystic Fibrosis
Cystic fibrosis is estimated to affect over 100,000 people worldwide, with an average life expectancy of only 50 years. Failed mucociliary clearance and mucus congestion in the lungs leads to cycles of infection and inflammation and an ongoing decline in lung function. Increasing fluid volume in the lung by inhibiting ENaC with ETD001 will hydrate mucus, improve clearance, reduce mucus congestion, and is expected to drive substantial improvements in lung function.
ETD001 has previously demonstrated a well-tolerated profile in healthy subjects in a phase 1 trial and has been shown to be long-acting in pre-clinical studies.
“We are thrilled to be granted the RPD (rare pediatric disease) designation by the FDA, a regulatory framework intended to encourage and accelerate innovative therapies, in recognition of the significance of our program in addressing an unmet medical need,” says Annabella Amatulli, head of regulatory affairs at Enterprise Therapeutics, in a release. “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 at the time of marketing approval.”
