384 results
-
List item
Orphan designation: Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment- for: Treatment in haematopoietic stem cell transplantation
Date of designation: 27/06/2016, Withdrawn, Last updated: 28/02/2022 -
List item
Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene for: The treatment of sickle cell disease
Date of designation: 29/04/2014, Withdrawn, Last updated: 07/12/2021 -
List item
Orphan designation: Ex-vivo cultured adult human mesenchymal stem cells for: Treatment of graft-versus-host disease
Date of designation: 20/02/2007, Positive, Last updated: 13/10/2021 -
List item
Orphan designation: Allogeneic ex-vivo-expanded umbilical cord blood-derived haematopoietic CD34+ progenitor cells and allogeneic non-expanded umbilical cord blood-derived haematopoietic mature myeloid and lymphoid cells for: Treatment in haematopoietic stem cell transplantation
Date of designation: 20/03/2017, Positive, Last updated: 13/10/2021 -
List item
Orphan designation: Allogeneic CD4+ and CD25+ T lymphocytes ex vivo incubated with GP120 for: Treatment in haematopoietic stem cell transplantation
Date of designation: 22/02/2018, Positive, Last updated: 17/09/2021 -
List item
Orphan designation: Heterologous human adult liver-derived stem cells for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of designation: 05/03/2012, Positive, Last updated: 26/08/2021 -
List item
Orphan designation: Heterologous human adult liver-derived stem cells for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 27/09/2011, Positive, Last updated: 26/08/2021 -
List item
Orphan designation: Heterologous human adult liver-derived stem cells for: Treatment of acute liver failure
Date of designation: 26/04/2012, Positive, Last updated: 26/08/2021 -
List item
Orphan designation: 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane (plerixafor) for: Treatment to mobilize progenitor cells prior to stem cell transplantation
Date of designation: 20/10/2004, Expired, Last updated: 10/08/2021 -
List item
Orphan designation: Allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (dilanubicel) for: Treatment in haematopoietic stem cell transplantation
Date of designation: 17/01/2018, Withdrawn, Last updated: 22/03/2021 -
List item
Orphan designation: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (darvadstrocel) for: Treatment of anal fistula
Date of designation: 08/10/2009, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: Ex-vivo-expanded autologous human corneal epithelium-containing stem cells for: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Date of designation: 07/11/2008, Positive, Last updated: 23/06/2020 -
List item
Orphan designation: Autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells for: Treatment of Asherman's syndrome
Date of designation: 20/04/2017, Positive, Last updated: 31/03/2020 -
List item
Orphan designation: Adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo for: Treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients
Date of designation: 16/01/2014, Withdrawn, Last updated: 12/02/2020 -
List item
Orphan designation: Human autologous bone-forming cells derived from bone marrow stem cells for: Treatment of non-traumatic osteonecrosis
Date of designation: 29/10/2007, Withdrawn, Last updated: 20/11/2019 -
List item
Orphan designation: carmustine for: Treatment in haematopoietic stem cell transplantation
Date of designation: 27/06/2018, Positive, Last updated: 21/08/2018 -
List item
Orphan designation: Human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor for: Treatment in haematopoietic stem cell transplantation
Date of designation: 12/01/2017, Positive, Last updated: 12/06/2018 -
List item
Orphan designation: 1-[[[4-(4-Fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride for: Treatment of soft tissue sarcoma
Date of designation: 22/02/2018, Positive, Last updated: 11/04/2018 -
List item
Orphan designation: Autologous adipose tissue-derived mesenchymal stem cells for: Treatment of thromboangiitis obliterans (Buerger's disease)
Date of designation: 20/03/2017, Positive, Last updated: 05/05/2017 -
List item
Orphan designation: Allogeneic bone marrow stem cells treated ex vivo with 16,16-dimethyl prostaglandin E2 for: Treatment of acute myeloid leukaemia
Date of designation: 13/05/2011, Withdrawn, Last updated: 07/02/2017 -
List item
Orphan designation: Allogeneic human adult stem cells, isolated from skeletal muscle and expanded ex vivo for: Treatment of Duchenne muscular dystrophy
Date of designation: 28/07/2015, Positive, Last updated: 18/08/2015 -
List item
Orphan designation: Allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells for: Prevention of bronchopulmonary dysplasia
Date of designation: 19/06/2015, Positive, Last updated: 27/07/2015 -
List item
Orphan designation: Allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus for: Treatment of adenovirus infection following haematopoietic stem cell transplantation
Date of designation: 19/03/2015, Positive, Last updated: 19/05/2015 -
List item
Orphan designation: Allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus for: Treatment of Epstein-Barr virus infection following haematopoietic stem cell transplantation
Date of designation: 19/03/2015, Positive, Last updated: 19/05/2015 -
List item
Orphan designation: Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a LAMB3-encoding retroviral vector for: Treatment of epidermolysis bullosa
Date of designation: 19/03/2015, Positive, Last updated: 11/05/2015