According to Krystal Biotech, the European Commission has granted orphan designation to the company’s KB407 inhaled gene therapy for the treatment of cystic fibrosis. The company said that KB407, which delivers two copies of the CFTR gene to the lungs via nebulization, also has received Orphan Drug designation from the FDA.
In August 2022, Krystal announced plans for a second Phase 1 trial of KB407 in CF patients. According to clinicaltrials.gov, that trial has not yet begun recruiting, and an earlier Phase 1 trial is still underway.
Krystal Biotech President of Research and Development Suma Krishnan commented, “We are pleased that the European Commission has granted orphan drug designation to KB407, which we believe is an important step toward advancing this option to treat patients with CF.”
Read the Krystal Biotech press release.
