CBER Regenerative Medicine Advanced Therapy (RMAT) Approvals
Data as of June 30, 2024
| Application Number | Submission Type and Number |
Proprietary Name | Established Name | Applicant | Approval Date | Use |
|---|---|---|---|---|---|---|
| BLA 125786 | ORIGINAL-1 | BEQVEZ | fidanacogene elaparvovec-dzkt | Pfizer, Inc. | 25-APR-2024 | Treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who are receiving routine prophylaxis and without pre-existing neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid detected by an FDA-approved test. |
| 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 |
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.