Long title
Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%)
Date receipt dossier
EU record number
B/BE/20/BVW6
EudraCT number
2019-004451-37
Company / Sponsor
Pfizer
Phase
III
Treated organism
Humans
Indication category
Genetic disorders
Disease
Adult males with moderately severe to severe Hemophilia A
Therapeutic approach
Gene therapy
Genetic modification
Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the human Factor VIII gene
Method of transfer of nucleic acid of interest
Non-replicating, non-integrating recombinant adeno-associated virus serotype 6 (AAV6)
Administered biological material
Recombinant virus
Type of procedure
Contained use and Deliberate release
Notes
Dossier withdrawn by the notifier on 7 December 2020 (before the advice of the Biosafety Council is finalised)