BioCentury
ARTICLE | Regulation

What’s in a name?

How FDA’s RMAT eligibility will benefit gene, CAR T therapies

September 15, 2017 12:42 AM UTC

FDA’s description of Kymriah tisagenlecleucel from Novartis AG as the “first gene therapy” approved in the U.S. provoked a debate about taxonomy that distracted from an important regulatory decision.

What matters is that FDA has decided certain gene therapies can be eligible for the Regenerative Medicine Advanced Therapy (RMAT) designation. Defining CAR T as gene therapy means they could qualify for RMAT designation, potentially speeding their development. ...