Sarepta Therapeutics has recently reported that they secured the very first Platform Technology Designation from the FDA. This recognition means the FDA views Sarepta’s delivery system a broader toolkit, not a one-off therapy. It allows Sarepta to leverage previously accepted data (e.g., manufacturing processes, safety, biodistribution) from Elevidys across multiple new gene therapy programs using the same AAV vector. That opens the door for streamlined regulatory review for future products built on the same platform.
It’s clear there’s growing momentum, both from innovators and regulators, to rethink how we develop and approve ATMPs. However, this particular designation applies solely to Sarepta and its proprietary vector. While a notable advancement, it also raises an important question for the broader field: How can platform technologies be made more widely accessible to developers in academia, start-ups, and public–private consortia?
If platform approaches are to truly accelerate development across the rare disease landscape, shared access and public infrastructure for vector production and validation will need to be developed.
This designation is an important step forward, but the field must now consider how to make sure that these advances benefit the wider therapeutic development space.
Please share your thoughts!