FDA Fast-tracks the First Inhalable Gene Therapy for Cancer
Early clinical findings have led the FDA to grant RMAT designation to a first-of-its-kind inhalable gene therapy being developed for advanced or metastatic non-small cell lung cancer.
Krystal Biotech, Inc. announced on Feb. 9 that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707, its redosable immunotherapy engineered to induce sustained, localised expression of interleukin-2 and interleukin-12 within the tumor microenvironment. The designation applies to the treatment of patients with advanced or metastatic non-small cell lung cancer (NSCLC).
Suma Krishnan, President of Research and Development at Krystal Biotech, said:
“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.”
The FDA’s RMAT designation is designed to facilitate and accelerate the development of regenerative medicine therapies, including gene therapies. To qualify, an investigational therapy must be intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, with preliminary clinical evidence suggesting the potential to address unmet medical needs. RMAT designation confers all benefits associated with the FDA’s Fast Track and Breakthrough Therapy programs, including eligibility for rolling review, increased regulatory interaction and guidance, and organisational commitment from senior FDA leadership. The pathway also allows for more frequent discussions with the FDA regarding innovative clinical trial designs, the use of surrogate or intermediate endpoints to support potential accelerated approval, and approaches to fulfilling post-approval requirements.
KYANITE-1 study Provides Supporting Clinical Evidence
Data supporting the RMAT designation were derived from early clinical findings in the ongoing KYANITE-1 study. The study demonstrated consistent and meaningful antitumor activity in patients with heavily pre-treated advanced NSCLC treated with inhaled KB707, including durable responses and clinically significant tumor reductions. Enrollment in KYANITE-1 is ongoing, and further details will be presented at upcoming scientific conferences. Additional details about the study can be found at www.clinicaltrials.gov under NCT identifier NCT06228326.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated, commercial-stage, global biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK®, the Company’s first commercial product, is the first-ever redosable gene therapy and the first genetic medicine approved in the United States, Europe, and Japan for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter).
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