While there has been a consistent stream of RMAT approvals since the designation was created in 2017, with 60 publicly announced to date, the number of approvals in the first few days in 2022 are noteworthy. Specifically, from January 1 to January 18, 2022, four companies publicly announced RMAT designations, as described below.
An RMAT designation is an award from 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.
RMAT Designations Accelerate
An RMAT designation is similar to the breakthrough therapy designation that is available to drugs. Recent RMAT awards from the first few days of 2022 are described below.
Allovir
On January 5th, 2022, Allovir announced it had received an RMAT designation for Posoleucel (Viralym-M, ALVR105) for the treatment of adenovirus (AdV) infections post-allogeneic stem cell transplantation.
Previously on June 11, 2019, Viralym-M (ALVR105) received an RMAT for the treatment of BK virus-associated hemorrhagic cystitis after HSC transplant.
As stated by said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer of AlloVir: “We are pleased that the FDA has granted posoleucel RMAT designation for a second treatment-related indication. The two RMAT designations reinforce the potential of posoleucel in areas of urgent patient need. We look forward to working closely with FDA as we continue to advance posoleucel through late-stage clinical development.”
Allovir is a late-clinical stage allogeneic T-cell immunotherapy company.
CARsgen Therapeutics
On January 10, 2022, CARsgen Therapeutics announced an RMAT designation for CT041 for patients with advanced gastric or gastroesophageal junction adenocarcinoma with Claudin18.2-positive tumor.
CT041 is CARsgen’s second product candidate with RMAT designation. Its first RMAT designation was received on October, 28, 2019, for CT053. CT053 is a CAR-T therapy used for the treatment of relapsed or refractory multiple myeloma.
As explained by Dr. Zonghai Li, Founder, Chairman of the Board, CEO and CSO of CARsgen Therapeutics: “The RMAT designation indicates that CT041 has the potential to solve the unmet medical needs of patients with GC/GEJ. RMAT is not only important for accelerating product development and the review of the biologics license application, but also helps patients obtain this advanced therapy as soon as possible. We hope that the RMAT and PRIME designations obtained by CT041 will enable us to work closely with the FDA and the EMA, to obtain approvals from regulatory agencies globally, and thus benefit patients worldwide as soon as possible.”
CARsgen develops novel CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors.
Cellular Biomedicine Group Inc (CBMG)
On January 12, 2022, Cellular Biomedicine Group Inc (CBMG) announced its RMAT designation for C-CAR039 for use in the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
As stated by Tony (Bizuo) Liu, Chairman and CEO of CBMG: “This is great news that the FDA has granted C-CAR039 both RMAT and fast track designations based on its potential to increase ORRs and CRs in relapsed/refractory DLBCL. The clinical data based on our clinical trials in China continue to support the hypothesis that C-CAR039 is the best-in-class CAR T asset for patients in this indication.”
Cellular Biomedicine Group, Inc. (NASDAQ: CBMG) develops novel cell therapies for the treatment of cancer and degenerative diseases.
ProKidney
On January 18, 2022, ProKidney announced its RMAT designation for REACT® (Renal Autologous Cell Therapy) for slowing the progression of Chronic Kidney Disease (CKD) and improving kidney function.
As stated by Tim Bertram, Founder and CEO of ProKidney: “Affecting more than 75 million patients in the United States and the European Union alone, CKD is one of the most challenging and burdensome chronic conditions to treat. For the first time, we have a multimodal approach to not only slow the onset of CKD, but in some cases reverse the loss of the kidney’s function. Through our advancements in cellular therapies, ProKidney can help usher in a new era of better health for millions of CKD patients living with the fear of kidney failure and a life on dialysis.
ProKidney is pioneering novel cellular therapies for the treatment of chronic kidney disease.
RMAT Awards in 2022
To date, 60 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced. However, the FDA states it has received 180 requests and issued 67, which means that a handful are not yet public knowledge.
Impressively, six companies have managed to receive two RMAT designations from the U.S. FDA, which is an impressive accomplishment. These companies are Abeona Therapeutics, AlloVir, Athersys, CARsgen Therapeutics, Rocket Pharmaceuticals, and CRISPR Therapeutics.
While the RMAT designation has existed since 2017, RMAT designations appear to be accelerating in momentum early this year in 2022.
Who do you think will be next on to receive one and for what product? Share your thoughts in comments below.
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