Notification report

Full notification file

General information

Notification Number

Member State to which the notification was sent

Date of acknowledgement from the Member State Competent Authority

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 in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions (Protocol number: 270-301)

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?

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

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:
09/01/2019 00:00:00