Krystal Biotech announced that the FDA has granted platform technology designation to the genetically modified, non-replicating herpes simplex virus type 1 (HSV-1) viral vector used in the company's investigational eye drop gene therapy KB801 for neurotrophic keratitis. The designation represents a significant regulatory milestone that could accelerate development of the company's genetic medicines pipeline.
Platform Technology Recognition
The FDA's platform technology designation program aims to provide efficiencies in drug development, manufacturing, and review processes for applications incorporating designated platform technologies. To qualify, a technology must be well-understood and reproducible, used in an FDA-approved product, and have potential to support multiple drug developments without compromising quality or safety.
"Receiving a platform technology designation from the FDA is a tremendous milestone for our development team and Krystal, both as recognition of the reproducibility and scalability of our HSV-1 gene delivery platform and for the potential product development benefits it may provide," said Suma Krishnan, President of Research and Development at Krystal Biotech.
Development Advantages
The designation may provide several benefits including early and more frequent FDA engagement during clinical development and opportunities to leverage manufacturing and nonclinical safety data from prior products using the platform technology, such as Krystal's approved VYJUVEK gene therapy. The FDA may also consider previous inspectional findings related to manufacturing of drugs incorporating the designated platform technology.
Krishnan noted the company is "excited to work with the FDA under this program to identify potential efficiencies, including opportunities to leverage our prior experience with FDA-approved VYJUVEK (beremagene geperpavec-svdt), to accelerate the development of our genetic medicines pipeline, starting with KB801 for the treatment of NK."
KB801 Gene Therapy Approach
KB801 is designed to enable sustained, localized expression and secretion of nerve growth factor (NGF) by epithelial cells in the front of the eye for treating neurotrophic keratitis, a rare degenerative corneal disease that leads to corneal epithelial defects, ulcers, and perforation.
Current recombinant NGF eye drops have demonstrated significant improvement in corneal healing and are approved for neurotrophic keratitis treatment in multiple jurisdictions worldwide. However, rapid clearance from the eye requires intensive administration six times daily, limiting therapeutic utility.
By enabling cells in the front of the eye to produce NGF locally, KB801 has potential to significantly reduce treatment burden for patients while maintaining more consistent NGF levels in the eye. The safety and efficacy of KB801 are currently under evaluation in EMERALD-1, a 2:1 randomized, double-masked, multicenter, placebo-controlled Phase 1/2 study in patients with neurotrophic keratitis.
Company Pipeline
Krystal Biotech is a commercial-stage biotechnology company focused on genetic medicines for 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 dystrophic epidermolysis bullosa treatment.
The company is advancing a pipeline of investigational genetic medicines across respiratory, oncology, dermatology, ophthalmology, and aesthetics applications.
