Platform Tech Designation: US FDA Emphasizes “Significant Efficiencies” Criteria In New Guidance

New draft guidance defines the necessary components of a platform technology designation request, including that sponsors demonstrate how it will save the FDA review time.

Hand is replacing part of a DNA molecule
Gene therapy is one area where the platform technology designation likely will be useful. • Source: Shutterstock

Sponsors seeking a platform technology designation from the US Food and Drug Administration should justify how the technology is reasonably likely to make the application review more efficient, a new agency draft guidance says.

Key Takeaways
  • The new platform technology designation program will require the FDA and industry to get comfortable with new terms, including for “platform technology.”

The guidance, released 28 May, seems to emphasize the requirement that a platform technology is likely to bring significant efficiencies...

More from Platform Technologies

More from Advanced Technologies

Elevidys Helps Sarepta To First US FDA Platform Technology Designation

 

Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.

HHS Secretary Favors Regenerative Medicine Over Traditional Pharma: Blessing And Curse?

 

Cell and gene therapy developers may not have to contend with HHS Secretary Robert F. Kennedy Jr.’s animosity like the traditional pharma industry, but his disinterest in distinguishing between the good and bad actors in the space could backfire.

Orphan Drug Trial Sponsors ‘Playing Catch-Up’ With Regulators – But Automation Can Help

 

Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.