Perspectives
Gene/Cell Therapy Quarterly Update: June 2022
July 13, 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 specialty drugs and traditional drugs.
New Drug Information
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New Drug information
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None
FDA APPROVED GENE/CELL THERAPY PRODUCTS
| 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
(liso-cel) |
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/CELL THERAPY 2022-2024 PIPELINE
| 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
(GS-010) |
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
20,000 |
|
| atidarsagene autotemcel
(OTL-200) |
Libmeldy™
|
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 | |
| ABO-102
(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 | |||||
| SB-525
(giroctocogene fitelparvovec) |
N/A | Sangamo/
Pfizer |
Hemophilia A | IV | 2024+ | 1,700 | |
| SRP-9001 (delandistrogene moxeparvovec) | N/A | Sarepta Therapeutics/
Roche |
Duchenne Muscular Dystrophy (DMD) | IV | 2024+ | 9,000-12,000 | |
| SRP-9003
(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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