Gene/Cell Therapy Quarterly Update: March 2023

Critical updates in an ever-changing environment

March 27, 2023

This 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/


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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