Krystal Biotech’s KB707 Receives RMAT Designation for Advanced Lung Cancer
Tuesday, February 10, 2026
Krystal Biotech has received Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA) for KB707, an investigational therapy for the treatment of advanced or metastatic non-small cell lung cancer (NSCLC).
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for around 80–85% of all cases. It develops from the epithelial cells of the lung and typically grows and spreads more slowly than small cell lung cancer.
KB707 is an inhaled, redosable immunotherapy designed to support sustained and localised expression of interleukin-2 and interleukin-12 within the tumour environment. The therapy is being developed for patients with advanced NSCLC, including those who have undergone multiple prior treatments.
The RMAT designation is granted to regenerative medicine therapies intended to treat serious or life-threatening conditions where early clinical evidence suggests the potential to address unmet medical needs.
This designation is aimed at accelerating development and review processes. It offers several regulatory advantages, including closer interaction with the FDA, guidance on trial design, eligibility for rolling review, and the possibility of using surrogate or intermediate clinical endpoints to support accelerated approval.
The designation for KB707 was supported by early clinical findings from the ongoing KYANITE-1 study. Preliminary results indicated meaningful anti-tumour activity in heavily pre-treated patients with advanced NSCLC, including tumour reduction and durable responses following inhaled administration of KB707.
Source: krystalbio.com
