Krystal Biotech Secures FDA RMAT Designation for KB707 in Advanced Lung Cancer
Tuesday, February 10, 2026
Krystal Biotech has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration for KB707, its redosable immunotherapy for the treatment of advanced or metastatic non-small cell lung cancer (NSCLC).
KB707 is designed to deliver sustained and localised expression of interleukin-2 and interleukin-12 within the tumour environment. The therapy is administered by inhalation and is being developed for patients with advanced stages of NSCLC.
The RMAT designation is intended to speed up the development of regenerative medicine therapies, including gene-based treatments, for serious or life-threatening conditions. To qualify, a therapy must show early clinical evidence suggesting it could address unmet medical needs. The designation offers several regulatory benefits, such as closer interaction with the FDA, intensive development guidance, and eligibility for rolling review and accelerated approval pathways.
The decision was supported by early clinical data from Krystal Biotech’s ongoing KYANITE-1 study. The study showed consistent anti-tumour activity in heavily pre-treated patients with advanced NSCLC, including durable responses and meaningful tumour reductions following treatment with inhaled KB707.
Patient enrolment in the KYANITE-1 study is continuing, and additional data are expected to be shared at upcoming scientific meetings. Further information about the trial is available on ClinicalTrials.gov under the identifier NCT06228326.
Source: globenewswire.com
