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Notification report


Full notification file


General information

Notification Number
B/IE/20/01

Member State to which the notification was sent
Ireland

Date of acknowledgement from the Member State Competent Authority
26/02/2020

Title of the Project
A Managed Access Program (MAP) Treatment Plan to provide AVXS-101 (onasemnogene abeparvovec) to any patient under the age of two with genetically confirmed SMA, regardless of type, symptom onset or prior treatment (AVXS-101-MAP-002).

Proposed period of release:
01/03/2020 to 31/12/2020

Name of the Institute(s) or Company(ies)
Children’s Hospital Ireland, Temple Street, Rotunda, Dublin 1;
Wellcome HRB Clinical Research Facility, St James’s Hospital, James’s street, Dublin 8;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Belgium; Germany; United Kingdom; Italy; Netherlands;

Has the same GMO been notified elsewhere by the same notifier?
No

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Family: Parvoviridae
Genus: Dependovirus
Species: Adeno-associated Virus (AAV9-derived replication-deficient viral vector containing human SMN cDNA)


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV2/9DependovirusAdeno-associated Virus--Serotype 2 and 9

European Commission administrative information

Consent given by the Member State Competent Authority:
Yes
13/03/2020 00:00:00
Remarks: