Fidanacogene elaparvovec

Fidanacogene elaparvovec (also known as SPK-9001 and BAX 335) is a gene therapy product that is under development for the treatment of hemophilia B. It is being developed by Spark Therapeutics and Pfizer.

Mechanism of action

Fidanacogene elaparvovec is a recombinant adeno-associated virus (AAV) vector-based gene therapy. It works by delivering a functional copy of the human factor IX (FIX) gene, which is mutated in people with hemophilia B, to the patient's liver cells. The delivered FIX gene then produces the FIX protein, which is necessary for blood clotting.

Clinical trials

Phase 1/2 clinical trials of fidanacogene elaparvovec have shown promising results, with patients showing sustained levels of FIX activity and a reduction in bleeding events. A phase 3 clinical trial is currently underway.

Regulatory status

Fidanacogene elaparvovec has been granted Orphan Drug designation by the Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) in the European Union.

See also

• Gene therapy
• Hemophilia B
• Adeno-associated virus
• Orphan Drug

External links

• ClinicalTrials.gov: Fidanacogene elaparvovec