FDA Grants First Platform Technology Designation to Sarepta’s Viral Vector

The Food and Drug Administration recently granted the first platform technology designation to Sarepta Therapeutics for AAVrh74 , the viral vector used in the company’s investigational gene therapy to treat limb-girdle muscular dystrophy type 2E/R4.

FDA defines a platform technology as a well-understood and reproducible technology, such as a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector or a combination of such technologies. To qualify, the technology must be essential to the structure or function of a product, be usable across multiple products with common structural elements and support standardized manufacturing.

For a technology to be designated, it must be incorporated into an approved drug or biologic and show potential to improve the efficiency of development, manufacturing and regulatory review for multiple products without compromising quality or safety. The designation enables sponsors to incorporate prior data generated with the platform to streamline the development and review of investigational new drug applications, new drug applications or biologic license applications.