FDA Grants Sarepta a Platform Technology Designation for Viral Vector

Rare Daily Staff

The U.S. Food and Drug Administration granted Sarepta Therapeutics platform technology designation for the rAAVrh74 viral vector used in its experimental gene therapy, SRP-9003, for the treatment of limb-girdle muscular dystrophy type 2E/R4.

The platform technology designation program is designed to help support rare disease therapies by streamlining drug development, manufacturing, and review processes for drug product applications, incorporating data from designated technologies.

To be eligible for this designation, the technology must be a well-understood and reproducible technology—such as a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies— and can support the development of multiple drugs or biologics. A platform is also eligible for consideration if it is incorporated in an approved drug and preliminary evidence demonstrates that the technology has the potential to be incorporated in or utilized by more than one drug without an adverse effect on quality, manufacturing, or safety, including when using the same manufacturing process.

Once granted, the designation enables sponsors to leverage prior data from the platform to support investigational new drug applications, new drug applications, or biologics license applications.

SRP-9003 is an experimental gene therapy designed to be systemically delivered to skeletal, diaphragm, and cardiac muscle, making it a well-suited candidate to treat neuromuscular diseases. SRP-9003 is designed to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to express in the heart, which is critically important for patients with limb-girdle muscular dystrophy type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

“This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,” said Louise Rodino-Klapac, chief scientific officer and head of research and development, Sarepta. “The designation underscores and reinforces the consistency of the data we have seen with this AAVrh74 in multiple clinical programs and is yet another example of Sarepta’s continued commitment to accelerating the development of potentially transformative treatments for patients with rare genetic diseases like LGMD type 2E/R4.”

Photo: Louise Rodino-Klapac, chief scientific officer and head of research and development, Sarepta