CBER Regenerative Medicine Advanced Therapy (RMAT) Approvals
Data as of June 30, 2023
| Application Number | Submission Type and Number |
Proprietary Name | Established Name | Applicant | Approval Date | Use |
|---|---|---|---|---|---|---|
| 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.