What's Happening?
Krystal Biotech has been granted the second-ever Platform Technology Designation by the FDA, following the revocation of Sarepta's designation earlier this year. This designation is intended to expedite
the approval process for therapeutics utilizing similar technology. Krystal's designation is based on a modified herpes simplex virus (HSV) vector, used in its approved topical therapy Vyjuvek for dystrophic epidermolysis bullosa. The company is currently testing KB801, an investigational drug for neurotropic keratitis, which shares the HSV vector. Analysts suggest that this designation could facilitate earlier and more frequent engagement with the FDA, potentially accelerating KB801's market entry by 2028.
Why It's Important?
The FDA's Platform Technology Designation is significant as it offers a streamlined path for therapeutics using established technology platforms, potentially reducing time to market. For Krystal Biotech, this could mean leveraging existing manufacturing and safety data from Vyjuvek for future submissions, enhancing efficiency in drug development. The designation underscores the FDA's commitment to supporting innovative treatments for rare diseases, which could benefit patients with limited therapeutic options. However, it also raises questions about the criteria for such designations, particularly concerning safety and efficacy across different administration routes.
What's Next?
Krystal Biotech may engage more frequently with the FDA to advance KB801 through clinical trials. The company could leverage its existing data from Vyjuvek to support the development of new applications using the HSV-1 vector. Stakeholders, including investors and healthcare providers, will be closely monitoring the progress of KB801 and other potential therapeutics under this designation. The FDA's approach to platform technology designations may evolve, influencing future drug development strategies.
Beyond the Headlines
The FDA's designation process highlights the balance between innovation and safety in drug development. As Krystal Biotech navigates the transition from topical to ocular applications, the FDA will scrutinize potential differences in tissue exposure and immune responses. This case may set precedents for how platform technologies are evaluated, impacting regulatory strategies for biotech companies.
