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
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.
|Asterias Biotherapeutics||AST-OPC1||Spinal Cord Injury (SCI)||asteriasbiotherapeutics.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|
|jCyte||jCell||Retinitis Pigmentosa (RP)||jcyte.com||2017|
|Juno Therapeutics||JCAR017||Lymphoma (large B cell NHL)||junotherapeutics.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
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 21st 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).
To learn more, view the “Global Regenerative Medicine Industry Database 2018.”
Who do you expect to appear next on this RMAT list? Mention them in the comments below.