Marketing authorisations for GMO-medicinal products for human use
| Fluenz (now replaced by Fluenz Tetra) |
| Application number |
EMEA/H/C/001101 |
| Marketing authorisation holder |
MedImmune LLC |
| Name of the product |
Fluenz |
| Active substances |
Influenza virus reassortants |
| Parental organism |
Live attenuated strain of Influenza virus |
| Transgene(s) |
Influenza virus reassortants for each of the 3 strains selected for the yearly season |
| Treated organism |
Humans: children en adolescents |
| Indication(s) |
Vaccination against influenza A (subtypes H1N1 and H3N2) and influenza B |
| Administration route(s) |
Nasal spray |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advices of the Biosafety Advisory Council |
08/04/2009, 17/06/2010, 13/09/2010 |
| EMA (CHMP) opinion (date) |
Positive (27/10/2010) |
| European Commission authorisation number (date) |
EU/1/10/661 (27/01/2011) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Glybera |
| Application number |
EMEA/H/C/2145 |
| Marketing authorisation holder |
uniQure biopharma B.V. |
| Name of the product |
Glybera |
| Active substances |
Viral vector |
| Parental organism |
Protein shell deived from adeno-associated virus serotype 1 |
| Transgene(s) |
Human lipoprotein lipase |
| Treated organism |
Humans: adults |
| Indication(s) |
Lipoprotein lipase deficiency (LPLD) |
| Administration route(s) |
Intramuscular |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advices of the Biosafety Advisory Council |
30/04/2010, 10/01/2011, 09/05/2011 |
| EMA (CHMP) opinion (date) |
Positive (19/07/2012) |
| European Commission authorisation number (date) |
EU/1/12/791 (25/10/2012) - with restricted access programme |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Imlygic |
| Application number |
EMEA/H/C/002771 |
| Marketing authorisation holder |
Amgen Europe B.V. |
| Name of the product |
Imlygic |
| Active substances |
Talimogene laherparepvec (oncolytic virus) |
| Parental organism |
Herpes simplex virus 1 |
| Transgene(s) |
Immune stimulatory protein human GM-CSF |
| Treated organism |
Humans: adults |
| Indication(s) |
Treatment of melanomas |
| Administration route(s) |
Injection into melanoma tumours |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
Not assesed by the Council |
| EMA (CHMP) opinion (date) |
Positive (22/10/2015) |
| European Commission authorisation number (date) |
EU/1/15/1064 (16/12/2015) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
B/BE/14/BVW1, B/BE/15/BVW1 (links to the Belgian Biosafety Server) |
| Pandemic Influenza vaccine H5N1 AstraZeneca |
| Application number |
EMEA/H/C/003963 |
| Marketing authorisation holder |
AstraZeneca AB |
| Name of the product |
Pandemic Influenza vaccine H5N1 Astra Zeneca |
| Active substances |
Pandemic Influenza vaccine H5N1 (live attenuated, nasal) |
| Parental organism |
Live attenuated influenza A virus A/Vietnam/1203/2004 (H5N1) strain |
| Transgene(s) |
|
| Treated organism |
Humans: children and adolescents |
| Indication(s) |
Prophylaxis of influenza in an officially declared pandemic situation in children and adolescents from 12 months to less than 18 years of age |
| Administration route(s) |
Intranasal route |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
None |
| EMA (CHMP) opinion (date) |
Positive (01/04/2016) |
| European Commission authorisation number (date) |
EU/1/16/1089 (20/05/2016) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Zalmoxis |
| Application number |
EMEA/H/C/2801 |
| Marketing authorisation holder |
MolMed S.p.A. |
| Name of the product |
Zalmoxis |
| Active substances |
Allogeneic T cells |
| Parental organism |
T-cells obtained from haploidentical donors |
| Transgene(s) |
Truncated form of the human low affinity nerve growth factor receptor and the herpes simplex I virus thymidine kinase |
| Treated organism |
Humans: adults |
| Indication(s) |
Adjunctive treatment in haploidentical haematopoietic stem cell transplantation (HSCT) of adult patients with high-risk haematological malignancies |
| Administration route(s) |
Intravenous infusion following cell thawing |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advices of the Biosafety Advisory Council |
11/03/2015, 12/01/2016, 21/03/2016 |
| EMA (CHMP) opinion (date) |
Positive (24/06/2016) |
| European Commission authorisation number (date) |
EU/1/16/1121 (18/08/2016) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Strimvelis |
| Application number |
EMEA/H/C/003854 |
| Marketing authorisation holder |
GlaxoSmithKline Trading Services Limited |
| Name of the product |
Strimvelis |
| Active substances |
autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence |
| Parental organism |
CD34+ cells extracted from the patient’s bone marrow |
| Transgene(s) |
Human adenosine deaminase (ADA) |
| Treated organism |
Humans: adults |
| Indication(s) |
Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advices of the Biosafety Advisory Council |
08/09/2015, 15/02/2016 |
| EMA (CHMP) opinion (date) |
Positive (01/04/2016) |
| European Commission authorisation number (date) |
EU/1/16/1097 (26/05/2016) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Kymriah |
| Application number |
EMEA/H/C/004090 |
| Marketing authorisation holder |
Novartis Europharm Limited |
| Name of the product |
Kymriah |
| Active substances |
Tisagenlecleucel, autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19. |
| Parental organism |
Autologous T cells |
| Transgene(s) |
Chimeric antigen receptor directed against CD19 |
| Treated organism |
Humans: adults |
| Indication(s) |
Paediatric and young adult patients up to 25 years of age with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post transplant or in second or later relapse. Adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
01/03/2018, 18/05/2018 |
| EMA (CHMP) opinion (date) |
Positive (28/06/2018) |
| European Commission authorisation number (date) |
EU/1/18/1297 (22/08/2018) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Yescarta |
| Application number |
EMEA/H/C/004480 |
| Marketing authorisation holder |
Kite Pharma EU B.V |
| Name of the product |
Yescarta |
| Active substances |
Axicabtagene ciloleucel, autologous T cells genetically modified to express a chimeric antigen receptor against CD19 |
| Parental organism |
Autologous T cells |
| Transgene(s) |
Chimeric antigen receptor targeting CD19 |
| Treated organism |
Humans: adults |
| Indication(s) |
Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
30/11/2017, 06/04/2018, 20/06/2018 |
| EMA (CHMP) opinion (date) |
Positive (28/06/2018) |
| European Commission authorisation number (date) |
EU/1/18/1299 (23/08/2018) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Luxturna |
| Application number |
EMEA/H/C/004451 |
| Marketing authorisation holder |
Novartis Europharm Limited |
| Name of the product |
Luxturna |
| Active substances |
Voretigene neparvovec, recombinant adeno-associated virus serotype 2 harbouring the human retinal pigment epithelium protein gene (RPE 65) |
| Parental organism |
Replication defective recombinant adeno-associated virus serotype 2 |
| Transgene(s) |
Human retinal pigment epithelium-specific 65 kDA protein gene (RPE65) |
| Treated organism |
Humans: adult and paediatric patients |
| Indication(s) |
Treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells |
| Administration route(s) |
Subretinal injection |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
27/11/2017, 30/04/2018, 11/07/2018 |
| EMA (CHMP) opinion (date) |
Positive (20/09/2018) |
| European Commission authorisation number (date) |
EU/1/18/1331 (22/11/2018) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Dengvaxia |
| Application number |
EMEA/H/C/004171 |
| Marketing authorisation holder |
Sanofi Pasteur |
| Name of the product |
Dengvaxia |
| Active substances |
Dengue tetravalent vaccine (live, attenuated) |
| Parental organism |
Attenuated yellow fever vaccine strain |
| Transgene(s) |
Each chimeric yellow fever dengue virus contains the prM and E genes from each of the dengue virus serotypes 1,2,3 and 4 |
| Treated organism |
Humans: adults |
| Indication(s) |
Prevention of dengue disease caused by dengue virus serotypes 1, 2, 3 and 4 in individuals 9 to 45 years of age with prior dengue virus infection and living in endemic areas |
| Administration route(s) |
Subcutaneous route |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
24/05/2015, 31/01/2017, 31/03/2018 |
| EMA (CHMP) opinion (date) |
Positive (18/10/2018) |
| European Commission authorisation number (date) |
EU/1/18/1338 (12/12/2018) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Zynteglo |
| Application number |
EMA/H/C/003691 |
| Marketing authorisation holder |
Bluebird bio (Netherlands) B.V. |
| Name of the product |
Zynteglo |
| Active substances |
Genetically modified stem cells transduced with third generation, type I HIV-based lentiviral vector encoding the gene for beta-globin |
| Parental organism |
Autologous CD34+ haematopoietic stem cells |
| Transgene(s) |
beta-A-T87Q-globin gene |
| Treated organism |
Humans: children (from 12 years) and adults |
| Indication(s) |
Treatment against transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD) |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
14/01/2019, 15/03/2019 |
| EMA (CHMP) opinion (date) |
Positive (29/03/2019) |
| European Commission authorisation number (date) |
EU/1/19/1367; EU/3/12/1091 (04/06/2019) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
None |
| Zolgensma |
| Application number |
EMEA/H/C/004750 |
| Marketing authorisation holder |
Novartis Gene Therapies EU Limited |
| Name of the product |
Zolgensma |
| Active substances |
Onasemnogene abeparvovec |
| Parental organism |
Adeno-associated virus serotype 9 |
| Transgene(s) |
Survival motor neuron gene (SMN1-2) |
| Treated organism |
Human: babies |
| Indication(s) |
Treatment against Spinal muscular atrophy (SMA) |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
08/02/2019, 11/06/2019 |
| EMA (CHMP) opinion (date) |
Positive (27/03/2020) |
| European Commission authorisation number (date) |
EU/1/20/1443; EU/3/15/1509 (18/05/2020) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
B/BE/18/BVW6 |
| Vaxchora |
| Application number |
EMEA/H/C/003876 |
| Marketing authorisation holder |
Emergent Netherlands B.V. |
| Name of the product |
Vaxchora |
| Active substances |
Vibrio cholerae, strain cvd 103-hgr, live |
| Parental organism |
CVD 103-HgR V. cholerae |
| Transgene(s) |
Vibrio cholerae in which 94% deletion of the genes encoding the A1 subunit of cholera toxin (CT) |
| Treated organism |
Humans: adult and paediatric patients |
| Indication(s) |
Vaccintation against cholera |
| Administration route(s) |
Oral |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
10/05/2019, 04/10/2019 |
| EMA (CHMP) opinion (date) |
Positive (01/02/2020) |
| European Commission authorisation number (date) |
EU/1/20/1423 (01/04/2020) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Ervebo |
| Application number |
EMEA/H/C/004554 |
| Marketing authorisation holder |
Merck Sharp & Dohme B.V. |
| Name of the product |
Ervebo |
| Active substances |
Recombinant vesicular stomatitis virus (strain indiana) with a deletion of the envelope glycoprotein, replaced with the zaire ebolavirus (strain kikwit 1995) surface glycoprotein |
| Parental organism |
Vesicular stomatitis virus |
| Transgene(s) |
A surface glycoprotein from Zaire Ebola virus |
| Treated organism |
Humans: adult |
| Indication(s) |
Vaccination against Ebola virus |
| Administration route(s) |
Injection |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
20/05/2019, 05/09/2019, 01/10/2019 |
| EMA (CHMP) opinion (date) |
Positive (18/10/2019) |
| European Commission authorisation number (date) |
EU/1/19/1392 (11/11/2019) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
B/BE/19/BVW4 |
| ZABDENO |
| Application number |
EMEA/H/C/005337 |
| Marketing authorisation holder |
Janssen-Cilag International NV |
| Name of the product |
ZABDENO |
| Active substances |
Recombinant Adenovirus type 26 (Ad26) encoding the glycoprotein (GP) of the Ebola virus Zaire (ZEBOV) Mayinga strain |
| Parental organism |
Adenovirus type 26 |
| Transgene(s) |
Glycoprotein (GP) of the Ebola virus Zaire |
| Treated organism |
Humans: adult and paediatric patients |
| Indication(s) |
Vaccination against Ebola virus |
| Administration route(s) |
Intramuscular injection |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
07/02/2020 |
| EMA (CHMP) opinion (date) |
Positive (29/05/2020) |
| European Commission authorisation number (date) |
EU/1/20/1444 (01-07-2020) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| MVABEA |
| Application number |
EMEA/H/C/005343 |
| Marketing authorisation holder |
Janssen-Cilag International NV |
| Name of the product |
MVABEA |
| Active substances |
Recombinant Modified Vaccinia Ankara Bavarian Nordic Virus encoding the: Ebola virus Zaire (ZEBOV) Mayinga strain glycoprotein (GP); Ebola virus Sudan Gulu strain GP; Ebola virus Taï Forest strain nucleoprotein and the Marburg virus Musoke strain GP |
| Parental organism |
Modified Vaccinia Ankara (MVA) virus |
| Transgene(s) |
Glycoprotein (GP) of EBOV, Sudan ebolavirus (SUDV), and Marburg Marburgvirus (MARV), as well as the nucleoprotein (NP) of the Taï Forest ebolavirus (TAFV). |
| Treated organism |
Humans: adult and paediatric patients |
| Indication(s) |
Vaccination against Ebola virus |
| Administration route(s) |
Intramuscular injection |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
07/02/2020 |
| EMA (CHMP) opinion (date) |
Positive (29/05/2020) |
| European Commission authorisation number (date) |
EU/1/20/1445 (01-07-2020) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Libmeldy |
| Application number |
EMEA/H/C/005321 |
| Marketing authorisation holder |
Orchard Therapeutics (Netherlands) B.V. |
| Name of the product |
Libmeldy |
| Active substances |
GM CD34+ stem cell containing a copy of the ARSA gene |
| Parental organism |
Autologous CD34+ cells |
| Transgene(s) |
Human arylsulfatase A (ARSA) gene |
| Treated organism |
Humans: adult and paediatric patients |
| Indication(s) |
Treatment against metachromatic leukodystrophy (MLD) |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
06/03/2020 |
| EMA (CHMP) opinion (date) |
Positive (16/10/2020) |
| European Commission authorisation number (date) |
EU/1/20/1493, EU/3/07/446, 17-12-2020 |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Abecma |
| Application number |
EMEA/H/C/004662 |
| Marketing authorisation holder |
Bristol-Myers Squibb Pharma EEIG |
| Name of the product |
Abecma |
| Active substances |
Idecabtagene vicleucel, genetically modified T cells transduced with third generation, type I HIV-based lentiviral vector encoding an anti-BCMA02 antigen |
| Parental organism |
Autologous T lymphocyte cells |
| Transgene(s) |
Chimeric antigen receptor (CAR) targeting the BCMA expression on myeloma cells |
| Treated organism |
Humans: adult |
| Indication(s) |
Treatment against relapsed and refractory multiple myeloma |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
04/09/2020 |
| EMA (CHMP) opinion (date) |
Positive (25/06/2021) |
| European Commission authorisation number (date) |
EU/1/21/1539, EU/3/17/1863 (18-08-2021) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Breyanzi |
| Application number |
EMEA/H/C/004731 |
| Marketing authorisation holder |
Bristol-Myers Squibb Pharma EEIG |
| Name of the product |
Breyanzi |
| Active substances |
Lisocabtagene maraleucel, genetically modified T cells transduced with third generation, type I HIV-based lentiviral vector encoding the gene CD19 |
| Parental organism |
Autologous CD8+ and CD4+ T cell |
| Transgene(s) |
Chimeric antigen receptor (CAR) targeting CD19-expressing cells |
| Treated organism |
Humans: adult |
| Indication(s) |
Treatment against relapsed or refractory large B cell lymphoma |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status)- Orphan designation withdrawal : 20/05/2022 |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
28/10/2020 |
| EMA (CHMP) opinion (date) |
Positive (28/01/2022) |
| European Commission authorisation number (date) |
EU/1/22/1631 (04-04-2022) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Ad26_COV2_S COVID-19 Vaccine Janssen |
| Application number |
EMEA/H/C/005737 |
| Marketing authorisation holder |
Janssen-Cilag International NV |
| Name of the product |
Ad26_COV2_S COVID-19 Vaccine Janssen |
| Active substances |
Recombinant Adenovirus type 26 |
| Parental organism |
Adenovirus type 26 (Ad26) |
| Transgene(s) |
Modified SARS-CoV-2 S protein |
| Treated organism |
Humans: adult |
| Indication(s) |
Vaccination against COVID-19 virus |
| Administration route(s) |
Intramuscular injection |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
05/01/2021 |
| EMA (CHMP) opinion (date) |
Positive (11/03/2021) |
| European Commission authorisation number (date) |
EU/1/20/1525 (11-03-2021) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Skysona |
| Application number |
EMEA/H/C/003690 |
| Marketing authorisation holder |
bluebird bio (Netherlands) B.V. |
| Name of the product |
Skysona |
| Active substances |
elivaldogene autotemcel, genetically modified stem cells enriched to express the human adrenoleukodystrophy protein (ALDP) |
| Parental organism |
Autologous T lymphocyte cells |
| Transgene(s) |
Human adrenoleukodystrophy protein (ALDP) |
| Treated organism |
Humans: paediatric patients |
| Indication(s) |
Treatment against childhood cerebral adrenoleukodystrophy |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status ended on 19-Nov-21) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
08/01/2021 |
| EMA (CHMP) opinion (date) |
Positive (21/05/2021) |
| European Commission authorisation number (date) |
EU/1/21/1563, EU/3/12/1003, Autorisation on 16-07-2021, Withdrawal on 18-11-2021 |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Vaxzevria (Astrazeneca) |
| Application number |
EMEA/H/C/005675 |
| Marketing authorisation holder |
AstraZeneca AB |
| Name of the product |
Vaxzevria (Astrazeneca) |
| Active substances |
Recombinant, replication-deficient chimpanzee adenovirus (ChAdOx1) |
| Parental organism |
Chimpanzee adenovirus |
| Transgene(s) |
Spike (S) glycoprotein of severe acute respiratory syndrome-coronavirus-2 (SARS CoV-2) |
| Treated organism |
Humans: adult |
| Indication(s) |
Vaccination against COVID-19 virus |
| Administration route(s) |
Intramuscular injection |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
13/01/2021 |
| EMA (CHMP) opinion (date) |
Positive (29/01/2021) |
| European Commission authorisation number (date) |
EU/1/21/1529 (29-01-2021) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
B/BE/20/BVW2 |
| Carvykti |
| Application number |
EMEA/H/C/005095 |
| Marketing authorisation holder |
Janssen-Cilag International NV |
| Name of the product |
Carvykti |
| Active substances |
Ciltacabtagene autoleucel, genetically modified T cells transduced with third generation, type I HIV-based lentiviral vector encoding an anti-B-cell maturation antigen |
| Parental organism |
Autologous CD8+ and CD4+ T cell |
| Transgene(s) |
Chimeric antigen receptor (CAR) targeting B- cell maturation antigen (BCMA) |
| Treated organism |
Humans: adult |
| Indication(s) |
Cancer immocellular therapy against relapsed or refractory multiple myeloma |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
30/08/2021 |
| EMA (CHMP) opinion (date) |
Positive (24/03/2022) |
| European Commission authorisation number (date) |
EU/3/22/1648 (30-05-2022) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Roctavian (previously Roxavvy) |
| Application number |
EMEA/H/C/004749 |
| Marketing authorisation holder |
Biomarin International Limited |
| Name of the product |
Roctavian |
| Active substances |
Recombinant adeno-associated virus serotype 5 (AAV5) expressing the human coagulation factor VIII (FVIII) gene under the control of a liver-specific promoter |
| Parental organism |
AAV5 |
| Transgene(s) |
Human coagulation factor VIII (FVIII) gene under the control of a liver-specific promoter |
| Treated organism |
Humans: adult |
| Indication(s) |
Treatment against Hemophilia A with FVIII deficiency |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
17-04-2020 (advice issued for Roxavvy) |
| EMA (CHMP) opinion (date) |
Withdrawal of initial marketing authorisation applicaton on 04/11/2020; Positive (24/06/2022) |
| European Commission authorisation number (date) |
EU/1/22/1668 (24/08/2022) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
B/BE/19/BVW2 |
| Tecartus (previously KTE-X19) |
| Application number |
EMEA/H/C/005102 |
| Marketing authorisation holder |
Kite Pharma EU B.V. |
| Name of the product |
Tecartus |
| Active substances |
CD19-directed genetically modified autologous T-cell transduced with murine γ-retroviral vector encoding the gene for CD19 |
| Parental organism |
Autologous CD4+ and CD8+ cells |
| Transgene(s) |
Chimeric antigen receptor (CAR) targeting the CD19 expression on B-cell malignancies |
| Treated organism |
Humans: adult |
| Indication(s) |
Treatment against relapsed or refractory mantle cell lymphoma (MCL) |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
08-05-2020 |
| EMA (CHMP) opinion (date) |
Positive (16/10/2020) |
| European Commission authorisation number (date) |
EU/1/20/1492 (14/12/2020) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Upstaza |
| Application number |
EMEA/H/C/005352 |
| Marketing authorisation holder |
PTC Therapeutics International Limited |
| Name of the product |
Upstaza |
| Active substances |
Recombinant adeno-associated virus serotype 2 |
| Parental organism |
WT AAV2 |
| Transgene(s) |
Human cDNA encoding for aromatic L-amino acid decarboxylase enzyme (hAADC) |
| Treated organism |
Humans: adult |
| Indication(s) |
Treatment against aromatic L amino acid decarboxylase (AADC) deficiency |
| Administration route(s) |
Intracerebral infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
15-05-2020 |
| EMA (CHMP) opinion (date) |
Positive (20/05/2022) |
| European Commission authorisation number (date) |
EU/1/22/1653 (18/07/2022) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Qdenga |
| Application number |
EMEA/H/C/005155 |
| Marketing authorisation holder |
Takeda GmbH |
| Name of the product |
Qdenga |
| Active substances |
Four recombinant live attenuated dengue virus strains |
| Parental organism |
Dengue virus,TDV-2 strain |
| Transgene(s) |
Premembrane (prM) and envelope (E) |
| Treated organism |
Humans: adult and paediatric patients |
| Indication(s) |
Vaccination against dengue disease from 4 years of age |
| Administration route(s) |
Subcutaneous injection |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
11-10-2021 |
| EMA (CHMP) opinion (date) |
Positive (14/10/2022) |
| European Commission authorisation number (date) |
EU/1/22/1699 (05/12/2022) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Hemgenix |
| Application number |
EMEA/V/C/004827 |
| Marketing authorisation holder |
CSL Behring GmbH |
| Name of the product |
Hemgenix |
| Active substances |
Recombinant, replication-deficient adenovirus |
| Parental organism |
Human adenovirus serotype 5 (Ad5) |
| Transgene(s) |
Human coagulation factor IX (FIX) gene under the control of a liver-specific promoter |
| Treated organism |
Humans: adult |
| Indication(s) |
Treatment against Hemophilia B with FIX deficiency |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
30-06-2022 |
| EMA (CHMP) opinion (date) |
Positive (15/12/2022) |
| European Commission authorisation number (date) |
EU/1/22/1715 (20/02/2023) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
B/BE/18/BVW7 |
| Casgevy |
| Application number |
EMEA/H/C/005763 |
| Marketing authorisation holder |
Vertex Pharmaceuticals Incorporated |
| Name of the product |
Casgevy |
| Active substances |
Exagamglogene autotemcel (exa-cel) |
| Parental organism |
Autologous CD34+ human hematopoietic stem and progenitor cells (hHSPCs) |
| Transgene(s) |
Gene editing of erythroid-specific enhancer genetic locus of the BCL11A gene |
| Treated organism |
Humans: paediatric patients |
| Indication(s) |
Treatment against transfusion-dependent β-thalassemia (TDT) or sickle cell disease (SCD) |
| Administration route(s) |
Intravenous infusion |
| Type of authorisation |
Centralised at European level (orphan status) |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
04-05-2023 |
| EMA (CHMP) opinion (date) |
Positive (14-12-2023) |
| European Commission authorisation number (date) |
EU/1/23/1787 (09-02-2024) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
| Ixchiq |
| Application number |
EMEA/H/C/005797 |
| Marketing authorisation holder |
Valneva Austria GmbH |
| Name of the product |
Ixchiq |
| Active substances |
Strain of chikungunya virus that has been attenuated (weakened) |
| Parental organism |
chikungunya virus |
| Transgene(s) |
Live-attenuated vaccine derived by reverse genetics from a CHIKV strain. Attenuation was achieved by deleting amino acids in a part of a non‑structural protein. As compared to the parent strain, this genetic modification reduces replication capability of the modified virus in vivo. |
| Treated organism |
Humans (> 18 years) |
| Indication(s) |
Active immunisation for the prevention of disease caused by chikungunya virus (CHIKV) in individuals 18 years and older |
| Administration route(s) |
Intramuscular injection |
| Type of authorisation |
Centralised at European level |
| Legal frame |
Council regulation (EC) No 726/2004 and the environmental safety requirements laid down by European Directive 2001/18/EC, part C and the Royal Decree of 21/02/2005, article 27 |
| Competent authority |
EMA/European Commission |
| Belgian advisory body |
Biosafety Advisory Council |
| Date of the advice of the Biosafety Advisory Council |
19-01-2024 |
| EMA (CHMP) opinion (date) |
Positive (30-05-2024) |
| European Commission authorisation number (date) |
EU/1/24/1828/001 (28-06-2024) |
| European Commission decision |
Community register of medicinal products |
| European Public Assessment Report (including EMA opinion, Product information leaflet and Summary of product characteristics in all EU languages) |
EPAR (link to Web site of EMA) |
| Notifications to Belgian authorities while in Research and Development stage |
none |
