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CBER Regenerative Medicine Advanced Therapy (RMAT) Approvals

Data as of March 31, 2024

Application Number Submission Type
and Number
Proprietary Name Established Name Applicant Approval Date Use
BLA 125758 ORIGINAL-1 LENMELDY atridarsagene autotemcel Orchard Therapeutics (Europe) Ltd. 18-MAR-2024 Indicated for treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
BLA 125773 ORIGINAL-1 AMTAGVI Lifileucel Iovance Biotherapeutics, Inc. 16-FEB-2024 Treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor.
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



lisocabtagene maraleucel

Juno Therapeutics, a Celgene Company


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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