Drug Profile
Giroctocogene fitelparvovec - Pfizer/Sangamo Therapeutics
Alternative Names: AAV cDNA gene therapy - Sangamo Therapeutics; AAV cDNA hF8 gene therapy; AAV cDNA human Factor 8 gene therapy; AAV Factor 8 cDNA gene therapy; AAV Factor 8 cDNA in vivo gene therapy; PF 07055480; Recombinant AAV2/6 human Factor 8 gene therapy - Sangamo Therapeutics; Recombinant AAV2/6 human Factor VIII gene therapy - Sangamo Therapeutics; SB-525; SB-525 cDNA gene therapyLatest Information Update: 18 Mar 2024
Price :
$50
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At a glance
- Originator Sangamo BioSciences
- Developer Pfizer; Sangamo Therapeutics
- Class Antihaemorrhagics; Gene therapies
- Mechanism of Action Factor VIII replacements; Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Phase III Haemophilia A
Most Recent Events
- 13 Mar 2024 Sangamo Therapeutics and Pfizer announces intention to submit MAA for haemophilia A (IV) in early 2025
- 13 Mar 2024 Sangamo Therapeutics and Pfizer plans a BLA submission for haemophilia A (IV) in the early 2025
- 09 Dec 2023 Updated efficacy and adverse events data from the Phase-I/II Alta trial in Haemophilia A presented at the 65th American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2023)