In the table below you will find an overview of gene and cell therapy products approved by EMA and/or FDA, with the exception of cord blood stem cell products. This table will be updated on a yearly basis. For inconsistencies, please contact us.
| Brand Name | Classification | INN/description | Indication | Company | EMA/FDA approved | Month/Year approved |
|---|---|---|---|---|---|---|
| Imlygic™ | In vivo GTMP | Talimogene laherparepvec / HSV-1 oncolytic virus containing the cDNA of human GM-CSF | Advanced metastatic melanoma | Amgen | Yes/Yes | December 2015/ October 2015 |
| Luxturna™ | In vivo GTMP | Voretigene neparvovec / AAV2 vector containing human RPE65 cDNA | Leber congenital amaurosis | Spark Therapeutics | Yes/Yes | November 2018/December 2017 |
| Zolgensma™ | In vivo GTMP | Onasemnogene abeparvovec / AAV9 vector containing the SMN1 transgene | Spinal muscular dystrophy (SMA) | AveXis/ Novartis | Yes/Yes | May 2020/May 2019 |
| Upstaza™ | In vivo GTMP | Eladocagene exuparvovec / AAV2 vector containing the cDNA of human dopa decarboxylase (DCC) | Patients > 18 months with aromatic L-amino acid decarboxylase deficiencies | PTC Tx | Yes/No | July, 2022 |
| Roctavian™ | In vivo GTMP | Valoctocogene roxaparvovec /AAV5 vector containing the cDNA of the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ) | Adult patients with severe hemophilia A | BioMarin | Yes/No | August, 2022 |
| Hemgenix™ | In vivo GTMP | Etranacogene dezaparvovec / AAV5 vector containing the cDNA of human coagulation factor IX | Adult patients with hemophilia B | CSL Behring/ UniQure | No/Yes | November, 2022 |
| Strimvelis™ | Ex vivo GTMP | Autologous CD34+ cells transduced with a retroviral vector containing the ADA cDNA encoding human adenosine deaminase (ADA) | Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) | Orchard Tx | Yes/No | May 2016 |
| Zalmoxis™ | Ex vivo GTMP | Allogeneic T cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor (ΔLNGFR) and the herpes simplex I virus thymidine kinase (HSV-TK Mut2) | Add on treatment for patients who have received a haploidentical hematopoietic stem cell transplant | MolMed S.p.A. | Withdrawn/No | August 2016 |
| Kymriah™ | Ex vivo GTMP | Tisagenlecleucel / autologous CAR T cells directed to CD19 | Acute Lymphoblastic Leukemia (ALL) | Novartis | Yes/Yes | August 2018/August 2017 |
| Yescarta™ | Ex vivo GTMP | Axicabtagen ciloleucel / autologous CAR T cells directed to CD19 | Non-Hodgkin lymphoma: -Large B cell lymphoma -Follicular lymphoma | Kite Pharma | Yes/Yes | August 2018/October 2017 |
| Zynteglo™ | Ex vivo GTMP | Betibeglogene autotemcel / Lentiviral vector containing the cDNA of the human HBB gene. | Transfusion-dependent beta thalassemia | Bluebird Bio | Yes/Yes | May 2019/August 2022 |
| Libmeldy™ | Ex vivo GTMP | Atidarsagene autotemcel / Lentiviral vector containing the human ARSA cDNA encoding arylsulfatase A | Metachromatic Leukodystrophy (MLD) | Orchard Therapeutics | Yes/No | December 2020/ |
| Tecartus™ | Ex vivo GTMP | Brexucabtagene autoleucel / autologous CAR T cells directed to CD19 | Mantle Cell Lymphoma Acute Lymphoblastic Leukemia (ALL) | Kite Pharma | Yes/Yes | December 2020/July 2020 |
| Breyanzi™ | Ex vivo GTMP | Lisocabtagen maraleucel / autologous CAR T cells directed to CD19 | Relapsed or Refractory Large B-cell lymphoma Non-Hodgkin lymphoma Follicular lymphoma | Bristol Meyers Squibb | Yes/Yes | April 2022/February 2021 |
| Abecma™ | Ex vivo GTMP | Idecabtagene vicleucel / autologous CAR T -cellsyu76h | Relapsed or Refractory Multiple Myeloma | Celgene | Yes/Yes | August 2021/ March 2021 |
| Carvykti™ | Ex vivo GTMP | Ciltacabtagene autoleucel / autologous CAR T cells directed to B cell maturation antigen (BCMA) | Relapsed or Refractory Multiple Myeloma | Janssen Biotech | Yes/Yes | May 2022/February 2022 |
| Skysona™ | Ex vivo GTMP | Elivaldogene autotemcel / autologous CD34+ cells tranduced with retroviral vector containing The cDNA of the human ABCD1 | Early, Active Cerebral Adrenoleukodystrophy | Bluebird Bio | Withdrawn/Yes | September 2022 |
| Provenge™ | SCTMP | Sipuleucel-T / autologous dendritic cells | Metastatic castrate-resistant hormone-refractory prostate cancer | Dendreon Pharma | No/Yes | Withdrawn/April 2010 |
| Holoclar™ | SCTMP | Ex vivo expanded autologous human corneal epithelial cells containing stem cells | Patients with corneal damage due to (chemical) burns | Holostem Advanced Therapies | Yes/No | February 2015 |
| Spherox™ | SCTMP | Spheroids of human autologous matrix-associated chondrocytes | Symptomatic articular cartilage defects of the femoral condyle and the patella of the knee | CO.DON Ag | Yes/No | July 2017 |
| Alofisel™ | SCTMP | Darvadstrocel / Mesenchymal stem cells from fat tissue of adult donors | Complex perianal fistulas in patients with Crohn’s disease | Takeda Pharma | Yes/No | March 2018 |
| Ebvallo™ | SCTMP | Tabelecleucel / allogeneic, EBV-specific T-cells from matched donor | Second-line treatment for transplant recipients who develop, as a result of immunosuppression treatment, Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disease (PTLD) | Atara Biotherapeutics | Yes/No | October 2022 |
| GINTUIT™ | TEP | Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen | Topical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival conditions in adults (venous leg ulcers) | Organogenesis Inc. | No/Yes | March 2012 |
| MACI™ | TEP | Matrix-applied characterised autologous cultured chondrocytes | The repair of single or multiple symptomatic, full-thickness cartilage defects of the knee with or without bone involvement in adults | Vericel | Withdrawn/Yes | December 2016 |
| StrataGraft™ | TEP | Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen | Adults with thermal burns containing intact dermal elements for which surgical intervention is clinically indicated | Mallinckrodt plc | No/Yes | June 2021 |
| Rethymic™ | TEP | Human allogeneic processed thymus tissue-agdc | Pediatric patients with Congenital Athymia | Enzyvant | No/Yes | October 2021 |
Luxturna
Luxturna (voretigene neparvovec-rzyl) is currently the only approved gene therapy available in the US. It is the first medication approved for an inherited genetic disease ever. Luxturna was approved by the FDA at December 19, 2017 for the United States and it is currently under supervision at the EMA for approval in Europe. It is developed by Spark Therapeutics and outlicensed commercial rights outside the United States to Novartis.
Luxturna is used for patients with an inherited form of retinal dystrophy. Retinal dystrophy is an umbrella term for a wide range of progressive eye diseases. ‘Retinal’ means that the condition is related to the retina, which is the back layer of the eye which converts light into an understandable message to the brain. ‘Dystrophy’ is a degenerative condition. Retinal dystrophy causes progressive reduction or deterioration of vision which eventually can result in complete blindness. Luxturna can be used for patients who have mutations in the RPE65 gene in both chromosomes.
Luxturna is an adeno-associated virus type 2 (AAV2)-based treatment where the correct copy of the RPE65 gene is delivered without disturbing the genome. Luxturna is injected directly into the retina so it can infect the retinal cells. When RPE65 is expressed in those cells, it can perform its function and in principle, halt the progression of the disease.
Imlygic
Imlygic (talimogene laherparepvec) is a genetically modified herpes virus used to treat melanoma. Imlygic was approved by the FDA at October 27, 2015 and by the EMA at December 17, 2015. It was originally developed by BioVex and continued by Amgen after the acquisition of BioVex by Amgen in 2011. Although melanomas shrink due to Imygic, there is no statistically significant benefit in overall survival. In other words, Imlygic does not extend lifes of patients with melanoma. The lack of benefits in combination with the price tag of approximately $65,000 makes Imlygic unpopular to prescribe for doctors.
Imlygic is an oncolytic viral therapy with attenuated life herpes simplex virus type 1 (HSV-1). In HSV-1, two genes are removed and one gene is added. The genes who are removed originally code for the proteins infected cell protein 34.5 (ICP34.5) and infected cell protein 47 (ICP47). ICP34.5 blocks the response of healthy cells to stop replicating and die after viral infection. However, cancer cells lack this system. By removing ICP34.5, HSV-1 can not replicate and kill normal cells, but in cancer cells they can. ICP47 suppresses an immune response to viral infection. Removing ICP47 should trigger an immune response, in theory. A gene coding for granulocyte colony-stimulating factor (GM-CSF) is inserted to promote an immune response to cancer cells infected by Imlygic. Despite these efforts, clinical evidence for an immune response to cancer cells due to Imlygic is not available.
Note: this page is under construction. New gene and cell therapy products will be added soon.