4DMT Secures FDA RPDD For Aerosolized 4D-710
(RTTNews) - 4D Molecular Therapeutics, Inc. (FDMT) has announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) for the Company's product candidate aerosolized 4D-710 to treat cystic fibrosis (CF) lung disease.
David Kirn, M.D., Co-founder and CEO of 4DMT, emphasized the significance of this regulatory milestone and the urgent need for new therapeutic options for individuals with CF lung disease, especially those who do not qualify for existing disease-modifying therapies.
The company's 4D-710 can potentially treat a wide range of people with cystic fibrosis, irrespective of the specific cystic fibrosis transmembrane conductance regulator (CFTR) mutation, and is designed for aerosol delivery to achieve CFTR expression within lung airway epithelial cells.
The company also said that the Phase 1/2 AEROW clinical trial is evaluating 4D-710 in people with CF who are not eligible for or cannot tolerate the currently approved CFTR modulators.
Cystic fibrosis is an inherited, progressive disease caused by mutations in the CFTR gene, impacting the lungs, pancreas, and other organs, ultimately leading to respiratory failure.
