FDA Grants Platform Technology Designation To Krystal Biotech’s HSV-1 Viral Vector Supporting KB801 For Neurotrophic Keratitis Treatment

Krystal Biotech, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted a platform technology designation to the genetically modified, non-replicating herpes simplex virus type 1 (HSV-1) viral vector used in the company’s redosable eye drop gene therapy, KB801. KB801 is currently being evaluated in a randomized, placebo-controlled trial for the treatment of neurotrophic keratitis (NK).

Suma Krishnan, President of Research and Development at Krystal Biotech, described the designation as a major milestone, highlighting that it recognizes both the reproducibility and scalability of Krystal’s HSV-1 gene delivery platform and the potential advantages it may offer in product development. She noted that the company looks forward to working closely with the FDA under this program to explore opportunities for efficiencies, including leveraging experience from the previously FDA-approved VYJUVEK® (beremagene geperpavec-svdt), to accelerate the development of Krystal’s genetic medicines pipeline, beginning with KB801.

The FDA’s platform technology designation program is designed to streamline drug development, manufacturing, and regulatory review for products that incorporate recognized platform technologies. Benefits may include earlier and more frequent engagement with the FDA during clinical development, as well as the ability to use manufacturing and nonclinical safety data from prior products using the designated technology, such as VYJUVEK, in future submissions. The FDA may also consider previous inspectional findings related to the manufacture of drugs utilizing the designated platform.

To qualify for platform technology designation, a technology must be well-characterized and reproducible, have been used in an FDA-approved product, be capable of supporting the development of multiple drug or biologic products without compromising quality, manufacturing, or safety, and have the potential to bring significant efficiencies to the development, manufacturing, or regulatory review process. Once a drug product application is recognized as incorporating the designated platform technology, it can potentially benefit from the efficiencies provided under the program.