Slow Burn: US FDA RMAT Designations Surge While Breakthrough Pace Softens

The US FDA’s regenerative medicine advanced therapy designation is seeing its biggest year yet after a slower ramp-up than the more established breakthrough therapy pathway.

data increasing
RMAT designations are growing while Breakthrough Therapy designations appear to have passed their peak, according to Pink Sheet data. • Source: Shutterstock

The US FDA’s newest expedited review program, the regenerative medicine advanced therapy (RMAT) designation, is gaining momentum after a slower ramp-up than the agency’s older breakthrough therapy designation (BTD), the Pink Sheet US FDA Performance Tracker shows.

Key Takeaways
  • 2024 is shaping up to be a banner year for the US FDA’s regenerative medicine advanced therapy designation with totals already exceeding the previous annual record for designations by more than one-third.

  • Only 15% of novel biologics approved by CBER have held an RMAT designation since the program was enacted in late 2016, but recipients make up 60% of 2024 novel approvals so far

The FDA’s Center for Biologics Evaluation and Research has approved 11 novel biologics with an RMAT, starting with Bristol Myers Squibb Company’s Breyanzi (lisocabtagene maraleucel), approved 5 February 2021 for a third-line and later large B-cell lymphoma indication

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