Table 3.
PRIME Products
| Proprietary Name | Description | Therapeutic Area | Therapeutic Indication | Developer |
|---|---|---|---|---|
| SPK-9001 | adeno-associated viral vector containing factor IX gene variant | hematology | treatment of haemophilia B | Spark Therapeutics |
| BMN 270 | adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene | hematology | treatment of haemophilia A | BioMarin |
| AMT-060 | adeno-associated viral vector serotype 5 containing human factor IX gene | hematology | treatment of haemophilia B | uniQure |
| AVXS-101 | Adeno-associated viral vector serotype 9 containing the human SMN gene | neurology | treatment of pediatric patients diagnosed with spinal muscular atrophy type 1 | AveXis |
| DNX-2401 | adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain | oncology | treatment of recurrent glioblastoma in patients for which a gross total resection is not possible or advisable or who refuse further surgery | DNAtrix Therapeutics |
| ATA129 | allogeneic Epstein-Barr virus-specific cytotoxic T lymphocytes | hematology | treatment of patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disorder in the allogeneic hematopoietic cell transplant setting refractory to rituximab | Atara Biotherapeutics |
| Lentiglobin | autologous CD34+ hematopoietic stem cells transduced with lentiviral vector encoding the human βA-T87Q-globin gene | hematology | treatment of beta-thalassaemia major | Bluebird Bio |
| NY-ESO-1c259T | autologous CD4 and CD8 T cells transduced with lentiviral vector containing an affinity-enhanced T cell receptor to target the cancer-testis tumor antigen | oncology | treatment of HLA-A*0201, HLA-A*0205, or HLA-A*0206 allele-positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumor expresses the NY-ESO-1 tumor antigen | Adaptimmune |
| JCAR017 | autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor | oncology | treatment of relapsed/refractory diffuse large B cell lymphoma (DLBCL) | Juno Therapeutics |
| CTL019 | autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | oncology | treatment of pediatric patients with relapsed or refractory B cell acute lymphoblastic leukemia | Novartis |
| KTE-C19 | autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor |
oncology | treatment of adult patients with DLBCL who have not responded to their prior therapy or have had disease progression after autologous stem cell transplant (ASCT) | Kite Pharma |
Date of search: May 24, 2017. Source of information: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000660.jsp.