According to Krystal Biotech, the FDA has granted regenerative medicine advanced therapy (RMAT) designation to the company’s KB707 inhaled gene therapy for the treatment of non-small cell lung cancer. The company notes that the designation was partially based on early data from the ongoing KYANITE-1 Phase 1/2 trial in patients with lung cancer, which it says “demonstrated consistent and meaningful antitumor activity, including durable responses and clinically significant tumor reductions, in patients with heavily pre-treated advanced NSCLC receiving inhaled KB707.”
The company previously received an RMAT designation for a topical wound care product for dystrophic epidermolysis bullosa. In addition to KB707, Krystal’s pipeline includes inhaled gene therapies for the treatment of cystic fibrosis (KB407) and for the treatment of alpha-1 antitrypsin deficiency (KB408).
Krystal Biotech President of Research and Development Suma Krishnan commented, “The FDA’s decision to grant RMAT designation to KB707 reflects both the urgent unmet need for new NSCLC therapies as well as the promising early clinical evidence of efficacy we have observed with inhaled KB707 in patients with advanced NSCLC. This is the second RMAT designation granted to a Krystal program and, as such, we know first-hand the benefits that this designation can provide to accelerate development and shorten the path to a potential approval. We are excited to work closely with the FDA to maximize the potential impact of our KB707 program for patients with NSCLC.”
Read the Krystal Biotech press release
