Perspectives
Gene/Cell Therapy Quarterly Update: SEPTEMBER 2022
September 30, 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, biosimilar drugs and traditional drugs.
New Drug Information
- Zynteglo®(betibeglogene autotemcel): The FDA has approved Bluebird bio’s Zynteglo also known as beti-cel as a one-time gene therapy to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. Beta-thalassemia, a type of inherited blood disorder, reduces the levels of normal hemoglobin and red blood cells via mutations in the beta-globin subunit; this can ultimately cause the delivery of oxygen throughout the body to be at insufficient levels. The subsequently low levels of red blood cells can result in a number of health problems, such as dizziness, weakness, fatigue, bone abnormalities, and other more serious complications. Zynteglo established the efficacy based on the achievement of transfusion independence, which is the point at which the patient maintains a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. Across two clinical studies evaluating adults and kids with transfusion-dependent beta thalassemia, 89% of the 41 patients on Zynteglo achieved transfusion independence.1 Bluebird bio anticipates its launch in 2023 with a one-time price of $2.8 million per patient.2 The list price doesn’t include the cost of hospitalization required for patients receiving the therapy.
- Skysona® (elivaldogene autotemcel): The FDA approved bluebird bio’s Skysona (elivaldogene autotemcel) to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). Skysona is a one-time gene therapy that adds functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells. The accelerated approval was based on the 24-month Major Functional Disabilities (MFDs) in Phase 2/3 study ALD-102 and Phase 3 ALD-104. Patients treated with Skysona had a 72% likelihood of MFD-free survival at 24 compared to untreated patients who had only a 43% likeliood of MFD-free survival.3Another treatment option allogeneic hematopoietic stem cell transplant (allo-HSCT). Skysona will be available by the end of 2022 through a limited number of Qualified Treatment Centers in the US at a one-time wholesale acquisition cost of $3 million.
New Indications
- None
FDA APPROVED GENE/CELL THERAPY PRODUCTS
Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | Month Approved | |
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 (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 THERAPY 2022-2024 PIPELINE
Generic Name | Brand Name | Manu-facturer | Indication(s) | Route of Admin | Anticipated Filing date* |
etranacogene dezaparvovec | EtranaDez™ | CSL Behring | Hemophilia B | IV | PDUFA: 11/24/2022 |
valoctocogene roxaparvovec | Roctavian™ | BioMarin | Hemophilia A | IV | September 2022 |
SRP-9001 (delandistrogene moxeparvovec) | N/A | Sarepta Therapeutics/
Roche |
Duchenne Muscular Dystrophy (DMD) | IV | Fall 2022 |
eladocagene exuparvovec | Upstaza™ | PTC Therapeutics | Aromatic L-amino acid decarboxylase (AADC) deficiency | Intracerebral | 4Q2022 |
lenadogene nolparvovec
(GS-010) |
Lumevoq™ | GenSight Biologics | Leber Hereditary Optic Neuropathy | Intravitreal injection | 4Q2022 |
exagamglogene autotemcel (CTX-001) | N/A | Vertex | Beta-thalassemia and sickle cell disease | IV | 4Q2022 |
atidarsagene autotemcel
(OTL-200) |
Libmeldy™
|
Orchard | Metachromatic Leukodystrophy (MLD) | IV | 2022-2023 |
SPK-8011
(dirloctocogene samoparvovec) |
N/A | Sparks | Hemophilia A | IV | 1Q2023 |
lovotibeglogene autotemcel (lovo-cel) | N/A | Bluebird Bio | Sickle cell anemia | IV | 1Q2023 |
AGTC-501 (laruparetigene zosaparvovec) | N/A | AGTC | X-linked retinitis pigmentosa | Subretinal | 1H2023 |
fidanacogene elaparvovec | N/A | Roche/Pfizer | Hemophilia B | IV | 3Q2023 |
OTL-201 | N/A | Orchard | MPS IIIA | IV | 2H2023 |
UX111
(rebisuligene etiparvovec) |
N/A | Ultragenyx | MPS IIIA | IV | 1Q2024 |
LYS-SAF-302 (olenasulfligene relduparvovec) | N/A | Lysogene | MPS IIIA | Intracerebral | 2H2023 |
fordadistrogene movaparvovec | N/A | Pfizer | DMD | IV | 4Q2023 |
RP-L201 (marnetegragene autotemcel) | N/A | Rocket | Severe leukocyte adhesion deficiency type 1 (LAD-1) | IV | 1H2023 |
Fanconi Anemia | 2024+ | ||||
SB-525
(giroctocogene fitelparvovec) |
N/A | Sangamo/
Pfizer |
Hemophilia A | IV | 3Q2024 |
FLT180a
(verbrinacogene setparvovec) |
N/A | Freeline Therapeutics | Hemophilia B | IV | 2H2024 |
DTX-201
(peboctocogene camaparvovec) |
N/A | Novo Nordisk | Hemophilia A | IV | 2024+ |
SRP-9003
(bidridstrogene xeboparvovec) |
N/A | Sarepta | Limb girdle muscular dystrophy type 2E | IV | 2024+ |
AMT-130 | N/A | uniQure | Huntington’s disease | Intracerebral | 2024+ |
REFERENCES
- https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-treat-adult-and-pediatric-patients-beta-thalassemia-who
- https://www.fiercepharma.com/pharma/bluebirds-28m-gene-therapy-zynteglo-gets-fda-backing-beta-thalassemia
- https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-fda-accelerated-approval-skysonar-gene
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