Fidanacogene elaparvovec is a liver-specific adeno-associated virus (AAV) vector containing a codon-optimized human coagulation FIX gene that was investigated as a potential treatment for hemophilia B. Hemophilia B is a... Hemophilia B is a rare X-linked genetic disorder characterized by abnormal coagulation due to dysfunctional coagulation factor IX with a male incidence estimated to be 1 in 30,000 male births worldwide.
- Mechanism
- Curator reviewed · 2 references
Fidanacogene elaparvovec is a gene therapy designed to introduce a functional copy of a high activity variant of the Factor IX gene (FIX-R338L) into hepatocytes to address the cause of Hemophilia B.
Fidanacogene elaparvovec is a non-replicating recombinant AAV vector that utilizes the AAVRh74var capsid to deliver a functional human Factor IX transgene. The AAVRh74var capsid is derived from the Rh74 AAV, which is not known to cause disease in humans. AAVRh74var capsid is able to transduce hepatocytes, the natural site of Factor IX synthesis. The Factor IX gene present in fidanacogene elaparvovec is designed to reside predominately as episomal DNA within transduced cells. Expression of the transgene is driven by a liver-specific promoter, which results in tissue-specific expression. As a result, fidanacogene elaparvovec helps to restore circulating Factor IX procoagulant activity and hemostatic potential in patients with Hemophilia B, which may limit bleeding episodes and the need for exogenous Factor IX treatment.
- Primary indication
- In Canada and the US, fidanacogene elaparvovec is indicated for the treatment of adults (aged 18 years or older) with moderately severe to severe Hemophilia B (congenital Factor IX deficiency) who are negative for neutralizing...Curator reviewed · 10 structured indications
- First approval
- Canada, 2023 · United States, 2024
- Code names
- AAV8 HFIX19 · PF-06838435 · SPARK 101 · SPK-9001
Resolves to
What you can answer from here — as of June 15, 2026
Which drugs share a target with Fidanacogene elaparvovec, and which of those have an active Phase 3 trial?
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