RT’s Three Key Takeaways:
- FDA Designation: The investigational cystic fibrosis remedy ETD001 has been granted uncommon pediatric illness designation by the FDA, acknowledging its potential to deal with severe unmet medical wants in kids.
- Mechanism of Motion: ETD001 targets the sodium channel (ENaC) within the airway epithelium to extend mucus hydration and clearance, with the purpose of bettering lung operate in cystic fibrosis sufferers.
- Ongoing Scientific Trial: ETD001 is at the moment being examined in a section 2a trial, assessing its security and efficacy in cystic fibrosis sufferers who are usually not eligible for or are usually not receiving CFTR modulator remedy.
Enterprise Therapeutics Ltd, a biopharmaceutical firm targeted on growing therapies for respiratory illnesses, introduced its novel cystic fibrosis investigational remedy, ETD001, has been granted uncommon pediatric illness designation by the US Meals and Drug Administration (FDA).
ETD001 is a low molecular weight compound with first-in-class potential, which targets the sodium channel (ENaC) within the airway epithelium to extend the hydration and clearance of mucus.
Enterprise introduced the dosing of the primary particular person with cystic fibrosis in its section 2a trial of ETD001 in July. The trial goals to ship medical proof-of-concept and to evaluate the protection and efficacy of ETD001 within the ~10% of these with cystic fibrosis with the very best unmet medical want. The examine will assess lung operate in folks with cystic fibrosis who’re both ineligible for or are usually not receiving CFTR modulator remedy.
The uncommon pediatric illness designation was based mostly on the evaluation of cystic fibrosis as a severe or life-threatening illness, primarily affecting people aged from start to 18 years.
Potential of ETD001 for Cystic Fibrosis
Cystic fibrosis is estimated to have an effect on over 100,000 folks worldwide, with a median life expectancy of solely 50 years. Failed mucociliary clearance and mucus congestion within the lungs results in cycles of an infection and irritation and an ongoing decline in lung operate. Growing fluid quantity within the lung by inhibiting ENaC with ETD001 will hydrate mucus, enhance clearance, scale back mucus congestion, and is predicted to drive substantial enhancements in lung operate.
ETD001 has beforehand demonstrated a well-tolerated profile in wholesome topics in a section 1 trial and has been proven to be long-acting in pre-clinical research.
“We’re thrilled to be granted the RPD (uncommon pediatric illness) designation by the FDA, a regulatory framework meant to encourage and speed up revolutionary therapies, in recognition of the importance of our program in addressing an unmet medical want,” says Annabella Amatulli, head of regulatory affairs at Enterprise Therapeutics, in a launch. “The RPD designation will give Enterprise entry to precious incentives and assist from the FDA through the improvement of ETD001, together with the eligibility to request a Precedence Overview Voucher on the time of promoting approval.”
