In the past two week, the U.S. FDA has awarded three new RMAT designations, bringing the total number of awards to 19.
The new RMAT designations were awarded to:
- Nightstar Therapeutics: Gene therapy (NSR-REP1) for choroideremia treatment – June 14, 2018
- Caladrius Biosciences: CD34+ cell therapy (CLBS14) for refractory angina – June 19, 2018
- Voyager Therapeutics: Gene therapy (VY-AADC ) for Parkinson’s disease – June 22, 2018
Overview of RMAT Designations
Introduced by Congress in the 21st Century Cures Act in 2017, sponsors of cell and gene therapies can be awarded an RMAT designation if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it could address unmet medical needs.
The RMAT designation makes products eligible for an accelerated development and review of a marketing application, similar to that available to drugs that receive a breakthrough therapy designation. It includes the opportunity for increased meeting opportunities with the FDA, as well as early interactions to discuss potential surrogate or intermediate endpoints.
In 2017, the FDA awarded a total of 12 RMAT designations. Through June 2018, another seven have been awarded, bringing the total to 19.
To see a full list of RMAT designations, click here.
Accelerating Rate of RMAT Designations
With three of 19 total RMAT approvals happening within the past two weeks, the rate of RMAT approvals appears to be accelerating.
On June 19th, Caladrius released a statement saying:
“We are delighted and encouraged that the FDA has recognized our CD34+ cell therapy program with an RMAT designation. Refractory angina is a serious condition with high morbidity and no known effective treatments. We look forward to working with the FDA to define a path to registration for our therapy with the aim of providing expeditious treatment to patients suffering from this condition,” said David J. Mazzo, Ph.D., President and CEO of Caladrius.
On June 14th, Nightstar Therapeutics released a statement saying:
“Receiving RMAT designation for NSR-REP1 highlights the potential of this gene therapy to maintain and improve visual acuity in choroideremia,” Dave Fellows, CEO of Nightstar, said in the release. “This designation further underscores a recognition of the serious nature of choroideremia and the urgent need to develop new treatments for those affected by inherited retinal diseases that would otherwise lead to blindness. We look forward to working closely with the FDA to discuss the NSR-REP1 development program and to determine how we can accelerate the pathway for making NSR-REP1 available to choroideremia patients.”
On June 14th, Voyager Therapeutics released a statement saying:
“The RMAT designation was based on our Phase 1b clinical data with VY-AADC and represents an important milestone for the program and recognition of this gene therapy as a potential treatment for Parkinson’s disease,” said Robert Pietrusko, Pharm.D., Senior VP of Regulatory Affairs and QA at Voyager. “We look forward to working closely with the agency through the benefits of the RMAT designation for guidance on the development of VY-AADC including advice to generate evidence needed to support its potential approval in an efficient manner.”
To learn more about the industry, view the “Global Regenerative Medicine Industry Database 2018.”
Who do you think will apply for a RMAT designation next? Leave your thoughts in the comments below!