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 11 gene and cell therapy products available in the US approved for treatment of 16 indications. The table below presents cell therapies as red; gene therapies as blue.
|
Product/Developer |
Approved indication |
|
|
2017 |
Kymriah (tisagenlecleucel) |
Relapsed or refractory acute lymphoblastic leukemia |
|
Yescarta (axicabtagene ciloleucel) |
Large B cell lymphoma |
|
|
Luxturna (voretigene neparvovec-rzyl) |
Leber’s congenital amaurosis Retinitis pigmentosa |
|
|
2018 |
Kymriah (tisagenlecleucel) |
Large B cell lymphoma |
|
2019 |
Zolgensma (onasemnogene abeparvovedxiol) |
Spinal Muscular Atrophy (SMA) Type 1 |
|
2020 |
Tecartus (brexucabtageneciloleucel) |
Relapsed or refractory mantle cell lymphoma |
|
2021 |
Breyanzi (lisocabtagene maraleucel) |
Relapsed or refractory large B cell lymphoma |
|
Abecma (Idecabtagene vicleucel) |
Relapsed or refractory multiple myeloma |
|
|
Yescarta (axicabtagene ciloleucel) |
Follicular lymphoma |
|
|
Tecartus (brexucabtageneciloleucel) |
Relapsed or refractory adult B-cell lymphoblastic leukemia |
|
|
2022 |
Carvykti (ciltacabtagene autoleucel) |
Relapsed or refractory multiple myeloma |
|
Kymriah (tisagenlecleucel) |
Refractory follicular lymphoma |
|
|
Zynteglo (betibeglogene autotemcel) |
Beta-thalassemia |
|
|
Skysona (elivaldogene autotemcel) |
Cerebral adrenoleukodystrophy (CALD) |
|
|
Hemgenix (etranacogene dezaparvovecdrib) |
Hemophilia B |
|
2023 |
Elevidys (delandistrogene moxeparvovec-rokl) |
Duchenne muscular dystrophy |
|
Roctavian (valoctocogene roxaparvovec) |
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
—Hemophilia A
—Sickle cell anemia - Additional treatments for ophthalmological conditions
- Neurological conditions
- Hematological conditions
- Higher prevalence disease treatments
- Age-related wet macular degeneration
- Osteoarthritis (knee)