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
Phase 3 open-label study to evaluate efficacy, safety, and tolerability of FIX gene transfer with fidanacogene elaparvovec (PF-06838435) in pediatric male participants <18 years of age with moderately severe to severe hemophilia B (FIX:C≤2%) (BeneGene-3)

Proposed period of release:
01/03/2021 to 31/03/2027

Name of the Institute(s) or Company(ies)
Pfizer, Inc., 235 East 42nd Street, New York, NY 10017-5755, USA.;

3. Is the same GMO release planned elsewhere in the Community?
Germany; France; Italy;

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:
Genus: Dependoparvovirus
Species: AAVSpark100 - bioengineered adeno-associated viral vector derived from a naturally occurring AAV serotype (Rh74)

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
-DependoparvovirusAdeno-associated Virus-AAV-Spark100-

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known