Table 1 List of approved cell and gene therapies.
From: Equitable access to cell and gene therapies in South Africa: opportunities and hurdles
Treatment | Indication | Description | Delivery vector | Approval date | List price |
|---|---|---|---|---|---|
Gene modified cell therapies | |||||
Strimvelis | ADA-SCID | Autologous HSPCs, gene modified to express ADA | Gamma-retroviral | 2016 | EUR 594,000 |
Kymriah (tisagenleleucel) | B-ALL (paediatric) | Autologous T-cells, gene modified to express an anti-CD19 CAR | Lentiviral | 2017 | USD 475,000 |
Yescarta (axicabtagene ciloleucel) | DLBCL (relapsed/ refractory) | Autologous T-cells, gene modified to express an anti-CD19 CAR | Gamma-retroviral | 2017 | USD 373,000 |
Zalmoxis* (nalotimagene carmaleucel) | Adjunctive treatment for haploidentical HSCT | Allogeneic T-cells, gene modified to express a suicide gene (herpes simplex I virus thymidine kinase) and selection marker (LNGFR) | Gamma-retroviral | 2017 | EUR 130,000 (per infusion, up to 4) |
Zynteglo (betibeglogene autotemcel) | Beta-thalassaemia (transfusion dependant) | Autologous HSPCs, gene modified to express HBB | Lentiviral | 2019 | USD 1,800,000 |
Tecartus (brexucabtagene autoleucel) | Relapsed/refractory Mantle cell lymphoma | Autologous T-cells, gene modified to express an anti-CD19 CAR | Retroviral | 2020 | USD 373,000 |
Breyanzi (lisocabtagene maraleucel) | Relapsed/refractory DLBCL not otherwise specified, high-grade BCL, primary mediastinal BCL, FL grade 3B | Autologous T-cells, gene modified to express an anti-CD19 CAR | Lentiviral | 2021 | USD 428,363 |
Libmeldy | Metachromatic leukodystrophy | Autologous HSPCs, gene modified to express arylsulfatase A (ARSA) | Lentiviral | 2021 | Not known |
Abecma (idecabtagene vicleucel) | Relapsed/refractory multiple myeloma | Autologous T-cells, gene modified to express an anti-BCMA CAR | Lentiviral | 2021 | USD 419,500 |
In vivo delivered gene therapies | |||||
Glybera (alipogene tiparvovec) | Familial lipoprotein lipase deficiency (Hyperlipoproteinemia Type I) | Intra-muscular injection of the human lipoprotein lipase gene | AAV | 2012 | Withdrawn [40] |
Luxturna (voretigene neparvovec-rzyl) | Inherited blindness (biallelic mutations in RPE65) | Intra-ocular gene delivery of functional RPE65 | AAV | 2017 | USD 425,000 (per affected eye) |
Zolgensma (onasemnogene abeparvovec-xioi) | Spinal muscular atrophy | Intravenous infusion for gene delivery of functional SMN1 | AAV | 2019 | USD 2,100,000 |
