The 21st Century Cures Act has some important regenerative medicine language in it. One part of that was attempting to accelerate FDA review of promising investigation regenerative medicine therapies. The mechanism for this was a new designation called Regenerative Advanced Therapy, now renamed Regenerative Medicine Advanced Therapy (RMAT) Designation.
I don’t think anyone was sure how this would play out in terms of how quickly the FDA would give RMAT designations and to how many investigational products. Already it’s clear the FDA seems to be embracing the spirit of the Cures provisions as it has given out quite a few FDA granted RMAT designations and done so quickly.
The first that I could see was given to Humacyte, which has an artificial vessel research program (see image). The artificial vessels become colonized with endogenous stem cells after implantation.
Keep in mind that RMAT designation does not equal FDA-approval overall, but it should mean the investigational therapy has solid data behind it and is promising, together equaling a faster FDA review and then potentially in the future approval should future data be strong. If some of the RMAT therapies are ultimately proven to be safe and effective, and via RMAT they were able to get to patients and help them more quickly, with few failures amongst the RMAT group, then the RMAT regulatory experiment will be proven a big success.
Here are the approved RMATs I could find so far:
- Humacyte (Vascular Access for Hemodialysis)
- Enzyvant (DiGeorge syndrome)
- jCyte (Retinitis Pigmentosa)
There may be more already given, but not yet in the public domain. If you know of others please comment on the post to let us know and I’ll add them.
@Bob,
Yeah, hopefully it’s a good thing. We’ll find out in a few years one way or another more clearly. I think of it as a regulatory experiment. Good point on the clinics, but they may end up complaining if they don’t get RMATs that the new, speedier system is still unfair to them because it still requires good, solid data. Paul
This is good – right? It sounds like the first part of a move by the FDA to reform the language of approval for regenerative medicines. But importantly, RMAT provides no exclusions to the need for proof of safety and efficacy and clinical trial data (and the spectre of approval without Phase 3 is not going to happen here).
But is it simply lip service? Roll up. roll up, FDA is now accelerating stem cell therapies, just as the public demanded. Well if that impression is given then kudos to FDA, as dodgy clinics pushing non-approved therapies cannot hide behind the “patients need cures fast” banner anymore and this may help the public sort the credible therapies from those who really have no intention of proving efficacy.