Gene/Cell Therapy Quarterly Update: December 2022

Critical updates in an ever-changing environment

December 29, 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 specialtydrugs, biosimilar drugs and  traditional drugs. 

New Drug Information

  • Hemgenix® (etranacogene dezaparvovec-drlb): The U.S. Food and Drug Administration (FDA) has approved CSL Behring’s Hemgenix for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. Hemgenix is a one-time gene therapy product given as a single dose by IV infusion. In one study, patients who received Hemgenix had increases in factor IX activity levels, a decreased need for routine factor IX replacement prophylaxis, and a 54% reduction in annualized bleeding rate (ABR) compared to baseline.1 In a recent cost-effectiveness analysis of the drug, weighing health benefits against offset costs, the Institute for Clinical and Economic Review (ICER), an independent nonprofit research organization suggested that a fair price for the drug to be between $2.93 million and $2.96 million.2 Hemgenix has launched with a wholesale acquisition cost (WAC) of $3.5 million per one-time dose.

New Indications

  • None

References

  1. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b
  2. https://icer.org/news-insights/press-releases/icer-publishes-evidence-report-on-gene-therapies-for-hemophilia-a-and-b/
FDA APPROVED GENE/CELL THERAPY PRODUCTS
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved  
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 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 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  
EXTENDED GENE THERAPY 2023-2024 PIPELINE
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Filing date* Prevalence
valoctocogene roxaparvovec Roctavian™ BioMarin Hemophilia A IV PDUFA: 3/31/2023 1,700
SRP-9001 (delandistrogene moxeparvovec) N/A Sarepta Therapeutics/

Roche

Duchenne Muscular Dystrophy (DMD) IV PDUFA: 5/29/2023 9,000-12,000
exagamglogene autotemcel (CTX-001) N/A Vertex Beta-thalassemia and sickle cell disease IV 1Q2023 1,500 and

20,000

lovotibeglogene autotemcel (lovo-cel) N/A Bluebird Bio Sickle cell anemia IV 1Q2023 ~20,000
eladocagene exuparvovec Upstaza™ PTC Therapeutics Aromatic L-amino acid decarboxylase (AADC) deficiency Intracerebral 1H2023 300 in world
AGTC-501 (laruparetigene zosaparvovec) N/A AGTC X-linked retinitis pigmentosa Subretinal 1H2023 20,000 in US and Europe
lenadogene nolparvovec

(GS-010)

Lumevoq™ GenSight Biologics Leber Hereditary Optic Neuropathy Intravitreal injection 2H2023 6.5/million
OTL-201 N/A Orchard MPS IIIA IV 2H2023 ~1,000
LYS-SAF-302 (olenasulfligene relduparvovec) N/A Lysogene MPS IIIA Intracerebral 2H2023 ~1,000
atidarsagene autotemcel

(OTL-200)

Libmeldy™ Orchard Metachromatic Leukodystrophy (MLD) IV 2023 ~460
fidanacogene elaparvovec N/A Roche/Pfizer Hemophilia B IV 3Q2023 3.9-6.5/million
fordadistrogene movaparvovec N/A Pfizer DMD IV 4Q2023 9,000-12,000
RP-L201 (marnetegragene autotemcel) N/A Rocket Severe leukocyte adhesion deficiency type 1 (LAD-1) IV 1H2023 25-50/year severe
Fanconi Anemia 2024+ 6.25/million
UX111

(rebisuligene etiparvovec)

N/A Ultragenyx MPS IIIA IV 1Q2024 ~1,000
SB-525

(giroctocogene fitelparvovec)

N/A Sangamo/

Pfizer

Hemophilia A IV 3Q2024 1,700
FLT180a

(verbrinacogene setparvovec)

N/A Freeline Therapeutics Hemophilia B IV 2H2024 3.9-6.5/million
SRP-9003

(bidridstrogene xeboparvovec)

N/A Sarepta Limb girdle muscular dystrophy type 2E IV 2025 3.5/million
AMT-130 N/A uniQure Huntington’s disease Intracerebral 2H2025 15.2/million
  • Gene Therapy: One-time administration with anticipated WAC of > $750,000

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