What is an RMAT Designation and Who Has One?

Congress included several provisions related to cell and gene therapies in the 21st Century Cures Act, the most important of which is the RMAT designation. RMAT stands for “Regenerative Medicine Advanced Therapy” designation.

What is an RMAT Designation?

Sponsors of cell and gene therapies are now eligible to obtain an RMAT designation if their product is intended to treat serious or life-threatening diseases, and there is preliminary clinical evidence that it can address unmet medical needs.

In this article:

RMAT List
Regenerative Medicine Advanced Therapy (RMAT)
RMAT Definition
Advanced Therapy Reviews by The FDA
Future RMAT Designations

RMAT List

What products have received an RMAT designation? To date, 24 RMAT designations have been publicly announced (see table below).

However, the U.S. FDA Commissioner, Dr. Scott Gottlieb, stated on Twitter that additional RMATs have also been awarded, bringing the total closer to 30.

A list of publicly announced RMAT designations is shown below.

COMPANY	PRODUCT	INDICATION	WEBSITE	YEAR
Asterias Biotherapeutics	AST-OPC1	Spinal Cord Injury (SCI)	asteriasbiotherapeutics.com	2017
Athersys	MultiStem	Ischemic Stroke	athersys.com	2017
Bluebird Bio	LentiGlobin	Severe Sickle Cell Disease	bluebirdbio.com	2017
Cellvation Inc. (Fortress Biotech Co.)	CEVA101	Traumatic Brain Injury (TBI)	cellvation.com	2017
Humacyte, Inc	Humacyl	Vascular Access for Hemodialysis	humacyte.com	2017
Enzyvant	RVT-802	DiGeorge Syndrome	enzyvant.com	2017
jCyte	jCell	Retinitis Pigmentosa (RP)	jcyte.com	2017
Juno Therapeutics	JCAR017	Lymphoma (large B cell NHL)	junotherapeutics.com	2017
Kiadis Pharma	ATIR101	Leukemia	kiadis.com	2017
Mallinckrodt Pharmaceuticals	Stratagraft	Thermal Burns	mallinckrodt.com	2017
Mesoblast, Ltd.	MPC-150-IM	Heart Failure	mesoblast.com	2017
Vericel Corporation	Ixmyelocel-T	Dilated Cardiomyopathy	vcel.com	2017
Abeona Therapeutics	EB-101	Recessive RDEB	abeonatherapeutics.com	2018
Abeona Therapeutics	ABO-102	Sanfilippo Syndrome Type A (MPS IIIA)	abeonatherapeutics.com	2018
Audentes Therapeutics, Inc	AT132	X-linked Myotubular Myopathy (XLMTM)	audentestx.com	2018
AxoGen, Inc.	Avance® Nerve Graft	Peripheral nerve repair	axogeninc.com	2018
Caladrius Biosciences	CLBS14 (CD34+ cell therapy program)	Refractory angina	caladrius.com	2018
Capricor Therapeutics	CAP-1001	Duchenne muscular dystrophy (DMD)	capricor.com	2018
Cellerant Therapeutics, Inc.	Romyelocel-L (human myeloid progenitor cells)	Prevention of Iinfections During Neutropenia	www.cellerant.com	2018
MiMedx Group	AmnioFix® Injectable	Osteoarthritis (OA) of the knee	mimedx.com	2018
Nightstar Therapeutics	NSR-REP1	Choroideremia (progressive vision loss)	nightstartx.com	2018
Poseida Therapeutics	P-BCMA-101	Relapsed/Refractory Multiple Myeloma	poseida.com	2018
Rocket Pharmaceuticals	RP-L102	Relapsed/Refractory Multiple Myeloma	poseida.com	2018
Voyager Therapeutics	VY-AADC	Fanconi Anemia (FA)	voyagertherapeutics.com	2018

Regenerative Medicine Advanced Therapy (RMAT)

RMAT is an acronym for “Regenerative Medicine Advanced Therapy” designation. Introduced in December 2016 as part of the 21st Century Cures Act, an RMAT designation can shorten time to market for innovative medicines.

Products eligible for an RMAT designation include:

Cell therapies
Gene therapies
Therapeutic tissue engineering products
Human cell and tissue products
Combination products using such therapies or products

RMAT Definition

An RMAT is defined as an award by the U.S. FDA that allows for faster, more streamlined approvals of regenerative medicine products within the United States, such as cell and gene therapies, tissue engineering products, and combination products. RMAT designations make innovative products eligible for quicker development and review of a marketing application.

An RMAT designation is similar to the breakthrough therapy designation that is available to drugs. Benefits of an RMAT include increased opportunities to meet with FDA officials, as well as early meetings to discuss potential surrogate or intermediate endpoints.

21st Century Cures Act

The 21st Century Cures Act is a law that was passed by the 114th U.S. Congress in December 2016. The act authorize $6.3 billion in funding, most of which is allocated for the National Institutes of Health (NIH).

The bill, passed by both houses of Congress and signed into law by President Obama, puts the U.S. on the same playing field as other countries that support accelerated of innovative medicines. Other regions that have accelerated pathways include Japan, South Korea, and the EU.

The bill encourages late-stage development in regenerative medicine, recognizing the field’s potential to address severe unmet medical needs.

Advanced Therapy Reviews by The FDA

For more detail, the following is from the U.S. FDA website:

As described in Section 3033 of the 21^st Century Cures Act, a drug is eligible for regenerative medicine advanced therapy (RMAT) designation if:

The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

Future RMAT Designations

There is also chatter within the industry that Tigenix will apply for an RMAT designation for Cx601, a local administration of expanded adipose-derived stem cells (eASCs).