Drug Profile
Fidanacogene elaparvovec - Pfizer/Spark Therapeutics
Alternative Names: AAV8 factor IX gene therapy; AAV8 hFIX19; BEQVEZ; Haemophilia B gene therapy - Spark Therapeutics; PF-06838435; rAAV-Spark100-hFIX-Padua; SPK 9001; SPK-FIXLatest Information Update: 08 Jan 2024
Price :
$50
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At a glance
- Originator The Childrens Hospital of Philadelphia
- Developer Pfizer; Spark Therapeutics
- Class Antihaemorrhagics; Gene therapies
- Mechanism of Action Factor IX 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
- Registered Haemophilia B