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CBER Regenerative Medicine Advanced Therapy (RMAT) Approvals

Data as of December 31, 2023

Application Number Submission Type
and Number
Proprietary Name Established Name Applicant Approval Date Use
BLA 125788 ORIGINAL-1 LYFGENIA lovotibeglogene autotemcel bluebird bio, Inc. 08-Dec-2023 Treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events (VOEs).
BLA 125787 ORIGINAL-1 CASGEVY exagamglogene autotemcel Vertex Pharmaceuticals, Inc. 08-Dec-2023 For the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs)
BLA 125720 ORIGINAL-1 ROCTAVIAN valoctocogene roxaparvovec-rvox Biomarin Pharmaceutical, Inc. 29-Jun-2023 For the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA approved test.
BLA 125774 ORIGINAL-1 VYJUVEK beremagene geperpavec-svdt Krystal Biotech, Inc. 19-May-2023 Treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
BLA 125685 ORIGINAL-1 RETHYMIC Allogeneic processed Thymus Tissue Enzyvant Therapeutics GmbH 08-Oct-2021 Immune reconstitution in pediatric patients with congenital athymia.
BLA 125730 ORIGINAL-1 StrataGraft Allogeneic Keratinocyte Cell Line (NIKS), Seeded on Rat Collagen (BD) Conditioned with Human Dermal Fibroblasts (Clonetics) Stratatech Corporation 15-Jun-2021 Indicated to promote durable wound closure & regenerative healing in the treatment of adult patients with debrided thermal burns that contain intact dermal elements, and for which surgical intervention is clinically indicated.

BLA 125714

ORIGINAL-1

BREYANZI

lisocabtagene maraleucel

Juno Therapeutics, a Celgene Company

05-Feb-2021

Treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma after at least two prior therapies.

 

* RMAT designation was enacted in the 21st Century Cures Act on December 13, 2016.


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