The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug development and review process, particularly for rare diseases.
The FDA granted Sarepta Therapeutics its first publicly documented Platform Technology Designation for a viral vector, called rAAVrh74, that the company uses in its investigational gene therapies.
Draft guidance for the designation was issued in 2024 under the Biden administration. Drug makers that receive the designation can use a “platform technology” to make more than one drug or biologic, with the aim of streamlining reviews and creating predictability for new drug applications created using the same technologies.
The regulator’s official recognition of Sarepta’s platform represents “a game-changing move” for cell and gene therapies, Audrey Greenberg, a venture partner at the Mayo Clinic and biopharma commentator, wrote in a LinkedIn post on Wednesday evening. “This marks a critical shift,” she continued, “away from single-asset risk, toward shared components and repeatable systems.”
The designation is a “huge step toward modular, scalable development,” Greenberg added, and the FDA’s move on Wednesday paves the way for “a plug-and-play future for gene therapy.”
Analysts at William Blair agreed, writing in a Wednesday note to investors that the Platform Technology Designation encourages the use of “standardized platforms across drug development,” in turn “streamlining development of therapeutics.” The overall effect, they continued, is to “reduce the burden on developers and regulators.”
For Sarepta in particular, the designation is “favorable,” according to William Blair, as it should help the company “accelerate the development of its follow-on gene therapy programs” while also lowering early R&D expenses. According to the biotech’s announcement, the viral vector rAAVrh74, is being used in its investigational gene therapy for limb-girdle muscular dystrophy (LGMD). Importantly, as noted by Jeffries analysts in a Thursday morning note, rAAVrh74 is the same vector used by Sarepta’s already-approved Duchenne muscular dystrophy gene therapy Elevidys.
Sarepta is also using the vector platform for at least two other LGMD therapies, as per William Blair, but these assets will be made through a different manufacturing process. “It is unclear if the platform designation could be leveraged for these investigational candidates in the future,” the analysts wrote.
Wednesday’s designation comes as FDA leaders in recent weeks have expressed support for some regulatory flexibility for rare diseases and gene therapies. Commissioner Marty Makary, for instance, said in an April interview on the Megyn Kelly Show that the agency could determine if a drug candidate has a “plausible mechanism” when considering it for approval, especially for diseases affecting “a small number of people.”
If an investigational therapy “makes sense physiologically” and has a mechanism that is “scientifically plausible,” the FDA could then approve this treatment on “a conditional basis,” Makary said.
Makary has so far been consistent with his support for accelerating drug development for rare diseases, Jefferies said in an investor note on Wednesday. In a conference held by the firm, Makary emphasized the need to “build on the successes of creative regulatory pathways, lending support to surrogate endpoints,” the note read.
And on Tuesday, during an event organized by the National Organization for Rare Disorders, newly installed Center for Biologics Evaluation and Research director Vinay Prasad—who has often been critical of accelerated approvals—committed to giving rare disease patients rapid access to “products that are even small steps forward.”
