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Notification report


Full notification file


General information

Notification Number
B/ES/18/12

Member State to which the notification was sent
Spain

Date of acknowledgement from the Member State Competent Authority
12/06/2018

Title of the Project
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions (Protocol number: 270-302)

Proposed period of release:
01/01/2019 to 31/12/2019

Name of the Institute(s) or Company(ies)
BioMarin Pharmaceutical Inc., ;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Germany;

Has the same GMO been notified elsewhere by the same notifier?
Yes

If yes, notification number(s):


GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Identity of the GMO (genus and species)
Genus: Dependovirus,
Species: Adeno-associated virus/ serotype 5 (AAV 5)


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
Adeno-associated virusDependovirusAdeno‐Associated VirusSerotype 5--

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known