Perspectives
Gene/Cell Therapy Quarterly Update: December 2022
Critical updates in an ever-changing environment
December 29, 2022This 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
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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