Gene/Cell Therapy Quarterly Update: June 2022

July 13, 2022

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 specialty drugs and traditional drugs

New Drug Information

  • None

New Drug information

  • None

Generic Name Brand Name Manufacturer Indication(s) Route of Admin Month Approved
ciltacabtagene autoleucel (cilta-cel) Carvykti™ Johnson & Johnson (Janssen) R/R multiple myeloma CAR-T February 2022  
allogeneic processed thymus tissue adgc Rethymic™ Enzyvant Congenital athymia Surgical October 2021  
idecabtagene vicleucel (bb2121, ide-cel) Abecma™ Bristol-Myers Squibb and bluebird bio Multiple myeloma CAR-T 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  
Generic Name Brand Name Manufacturer Indication(s) Route of Admin Anticipated Filing date* Prevalence
betibeglogene autotemcel N/A Bluebird Bio Transfusion-dependent beta-thalassemia (TDT) IV PDUFA: 8/19/2022 1,500  
elivaldogene autotemcel Lenti-D™ Bluebird Bio Cerebral adrenoleukodystrophy (CALD) IV PDUFA: 9/16/2022 ~60  
etranacogene dezaparvovec EtranaDez™ CSL Behring Hemophilia B IV PDUFA: 11/24/2022 3.9-6.5/million


valoctocogene roxaparvovec Roctavian™ BioMarin Hemophilia A IV 2023 1,700  
eladocagene exuparvovec PTC-AADC PTC Therapeutics Aromatic L-amino acid decarboxylase (AADC) deficiency Intra-cerebral 2023 300 in world  
lenadogene nolparvovec


Lumevoq™ GenSight Biologics Leber Hereditary Optic Neuropathy Intra-vitreal injection 2023 6.5/million  
CTX-001 N/A Vertex Beta-thalassemia and sickle cell disease IV 2023 1,500 and


atidarsagene autotemcel




Orchard Metachromatic Leukodystrophy (MLD) IV 2023 ~460  
lovotibeglogene autotemcel (lovo-cel) N/A Bluebird Bio Sickle cell anemia IV 2023 ~20,000  
AGTC-501 (laruparetigene zosaparvovec) N/A AGTC X-linked retinitis pigmentosa Sub-retinal 2023 20,000 in U.S. and Europe  
fidanacogene elaparvovec N/A Roche/Pfizer Hemophilia B IV 2023 3.9-6.5/million  
OTL-201 N/A Orchard MPS IIIA IV 2024 ~1,000  

(rebisuligene etiparvovec)

N/A Ultragenyx MPS IIIA IV 2024 ~1,000  
LYS-SAF-302 (olenasulfligene relduparvovec) N/A Lysogene MPS IIIA Intra-cerebral 2024 ~1,000  
fordadistrogene movaparvovec N/A Pfizer DMD IV 2024 9,000-12,000  
RP-L201 (marnetegragene autotemcel) N/A Rocket Severe leukocyte adhesion deficiency type 1 (LAD-1) IV 2024 25-50/year severe  
      Fanconi Anemia   2024+ 6.25/million  

(giroctocogene fitelparvovec)

N/A Sangamo/


Hemophilia A IV 2024+ 1,700  
SRP-9001 (delandistrogene moxeparvovec) N/A Sarepta Therapeutics/


Duchenne Muscular Dystrophy (DMD) IV 2024+ 9,000-12,000  

(bidridstrogene xeboparvovec)

N/A Sarepta Limb girdle muscular dystrophy type 2E IV 2024+ 1:14,5000 to 123,000  
AMT-130 N/A uniQure Huntington’s disease Intra-cerebral 2024+ 1-5/100,000  

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