Approved cell and gene therapy (CGT) products

The FDA’s drug approval process is designed to ensure that drugs are safe and effective for their intended use before they are made available to the public. Within the FDA, the Center for Biologics Evaluation and Research (CBER) reviews and regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight. There are currently 19 gene and cell therapy products available in the US approved for treatment of 27 indications. The table below presents cell therapies as red; gene therapies as blue.

Product/Developer

Approved indication

2017

Kymriah (tisagenlecleucel)
Novartis

Relapsed or refractory acute lymphoblastic leukemia

Yescarta (axicabtagene ciloleucel)
Kite Pharma, Inc.

Large B cell lymphoma

Luxturna (voretigene neparvovec-rzyl)
Spark Therapeutics, Inc.

Leber’s congenital amaurosis

Retinitis pigmentosa

2018

Kymriah (tisagenlecleucel)
Novartis Pharmaceuticals

Large B cell lymphoma

2019

Zolgensma (onasemnogene abeparvovedxiol)
Novartis Pharmaceuticals

Spinal Muscular Atrophy (SMA) Type 1

2020

Tecartus (brexucabtageneciloleucel)
Kite Pharma, Inc.

Relapsed or refractory mantle cell lymphoma

2021

Breyanzi (lisocabtagene maraleucel)
Bristol-Myers Squibb

Relapsed or refractory large B cell lymphoma

Abecma (Idecabtagene vicleucel)
Bristol-Myers Squibb

Relapsed or refractory multiple myeloma

Yescarta (axicabtagene ciloleucel)
Kite Pharma, Inc.

Follicular lymphoma

Tecartus (brexucabtageneciloleucel)
Kite Pharma, Inc.

Relapsed or refractory adult B-cell lymphoblastic leukemia

2022

Carvykti (ciltacabtagene autoleucel)
Janssen Pharmaceuticals

Relapsed or refractory multiple myeloma

Kymriah (tisagenlecleucel)
Novartis Pharmaceuticals

Refractory follicular lymphoma

Zynteglo (betibeglogene autotemcel)
bluebird bio, Inc.

Beta-thalassemia

Skysona (elivaldogene autotemcel)
bluebird bio, Inc.

Cerebral adrenoleukodystrophy (CALD)

Hemgenix (etranacogene dezaparvovecdrib)
CSL Behring

Hemophilia B

2023

Elevidys (delandistrogene moxeparvovec-rokl)
Sarepta Therapeutics

Duchenne muscular dystrophy

Roctavian (valoctocogene roxaparvovec)
BioMarin

Hemophilia A

Casgevy (exagamglogene autotemcel)
Vertex

Sickle cell disease

Lyfgenia (lovotibeglogene autotemcel)
bluebird bio

Sickle cell disease

Vyjuvek (beremagene geperpaver-svdt)
Krystal Biotech, Inc.

Dystrophic epidermolysis bullosa

2024

Casgevy (exagamglogene autotemcel)
Vertex

Transfusion dependent Beta Thalassemia

Lenmeldy (atidarsagene autotemcel)
Orchard Therapeutics

Metachromatic leukodystrophy (MLD)

Breyanzi (lisocabtagne maraleucel)
Bristol-Myers Squibb

Relapsed or refractory chronic lymphocytic leukemia; small lymphocytic lymphoma; mantle cell lymphoma

Beqvez (fidanacogene elaparvovec)
Pfizer/Spark Therapeutic

Hemophilia A

NEWDIGS FoCUS maintains a pipeline analysis of clinical trials and FDA approvals of CGTs. Greater detail on therapies in the pipeline is detailed in the Pipeline Deep Dive. While clinical trial outcomes and regulatory approvals are never guaranteed, by 2026 we expect the following types of products may be available to patients:

  • Oncology
    • Additional therapies for B-cell leukemia and lymphoma
  • Rare disease treatments
    • Hematological conditions
    • Additional treatments for ophthalmological conditions
    • Neurological conditions
  • Higher prevalence disease treatments
    • Age-related wet macular degeneration
    • Osteoarthritis (knee)