Gene/Cell Therapy Quarterly Update: SEPTEMBER 2022

September 30, 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, 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
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


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 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/


Duchenne Muscular Dystrophy (DMD) IV Fall 2022
eladocagene exuparvovec Upstaza™ PTC Therapeutics Aromatic L-amino acid decarboxylase (AADC) deficiency Intracerebral 4Q2022
lenadogene nolparvovec


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




Orchard Metachromatic Leukodystrophy (MLD) IV 2022-2023

(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

(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+

(giroctocogene fitelparvovec)

N/A Sangamo/


Hemophilia A IV 3Q2024

(verbrinacogene setparvovec)

N/A Freeline Therapeutics Hemophilia B IV 2H2024

(peboctocogene camaparvovec)

N/A Novo Nordisk Hemophilia A IV 2024+

(bidridstrogene xeboparvovec)

N/A Sarepta Limb girdle muscular dystrophy type 2E IV 2024+
AMT-130 N/A uniQure Huntington’s disease Intracerebral 2024+


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