Perspectives
Gene/Cell Therapy Quarterly Update: March 2023
Critical updates in an ever-changing environment
March 27, 2023This quarterly pipeline wrap-up provides a review of newly approved gene and cell therapies, new indications and news of note on gene and cell therapies drugs in the approval process. See other articles for updates on specialtydrugs, biosimilar drugs and traditional drugs.
New Drug Information
- None
New Indications
- None
FDA approved Gene/Cell Therapy Products
| Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | Month Approved | |||
| nadofaragene firadenovec-vncg | Adstiladrin® | Ferring and FKD Therapies Oy | Certain forms of bladder cancer | Catheter into bladder | December 2022 | |||
| etranacogene dezaparvovec-drlb | Hemgenix™ | CSL Behring | Hemophilia B | IV | November 2022 | |||
| elivaldogene autotemcel | Skysona™ | Bluebird Bio | Cerebral adrenoleukodystrophy (CALD) | IV | September 2022 | |||
| betibeglogene autotemcel | Zynteglo® | Bluebird Bio | Transfusion-dependent beta-thalassemia (TDT) | IV | August 2022 | |||
| ciltacabtagene autoleucel | Carvykti™ | Johnson & Johnson (Janssen) | R/R multiple myeloma | CAR-T-cell therapy | February 2022 | |||
| allogeneic processed thymus tissue adgc | Rethymic™ | Enzyvant | Congenital athymia | Surgical | October 2021 | |||
| idecabtagene vicleucel | Abecma™ | Bristol-Myers Squibb and bluebird bio | Multiple myeloma | CAR-T-cell therapy | March 2021 | |||
| lisocabtagene maraleucel | Breyanzi™ | Bristol-Myers Squibb | Large B cell lymphoma (LBCL) | IV | February 2021 | |||
| brexucabtagene autoleucel | Tecartus™ | Kite | Mantle cell lymphoma | CAR T-cell therapy | July 2020 | |||
| onasemnogene abeparvovec-xioi | Zolgensma™ | AveXis | Spinal Muscular Atrophy | IV | May 2019 | |||
| voretigene neparvovec-rzyl | Luxturna® | Spark Therapeutics | Biallelic RPE65 mutation-associated retinal dystrophy | Subretinal injection | December 2017 | |||
| axicabtagene ciloleucel | Yescarta® | Kite | LBCL/follicular lymphoma (FL) | IV | October 2017 | |||
| tisagenlecleucel | Kymriah® | Novartis | Acute lymphoblastic leukemia/LBCL | IV | August 2017 | |||
Gene and Cell Therapy Pipeline
| Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | PDUFA date |
| omidubicel | N/A | Gamida Cell | Blood cancers in need of allogeneic hematopoetic stem cell transplant (HSCT) | Cell therapy | May 2023 |
| SRP9001 (delandistrogene moxeparvovec) | N/A | Sarepta Therapeutics/
Roche |
Duchenne Muscular Dystrophy (DMD) | IV | May 2023 |
| beremegene geperpavec | Vyjuvek® | Krystal Biotech | Dystrophic epidermolysis bullosa (DEB) | Topical | May 2023 |
| valoctocogene roxaparvovec | Roctavian™ | BioMarin | Hemophilia A | IV | June 2023 |
| lifileucel | N/A | Iovance | Unresectable or metastatic melanoma | CAR-T | 2Q2023 |
| remestemcel-L | N/A | Mesoblast | Children with steroid-refractory acute graft-versus-host disease | IV | July 2023 |
* Gene Therapy: One-time administration with curative intent and anticipated WAC of > $750,000
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