Fidanacogene elaparvovec

Unassigned

New Medicines

Haemophilia B

Information

Advanced therapy medicinal product (ATMP)
Pfizer
Pfizer

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes

Category

Single infusion of a gene therapy that uses an adeno-associated viral vector to deliver a codon-optimised, high-activity gene for human Factor IX to liver cells
Haemophilia B has a prevalence of around 1 in 30,000 live births (about five times rarer than haemophilia A). There are usually carrier females and affected males. The reference level of factor IX is 5 μg/mL but the normal range is from half to twice that level. Severe disease occurs with a factor IX level below 1% of the reference and accounts for about 50% of cases. Moderate severity occurs with a level of 1-5% and accounts for around 30% of cases [1].
Haemophilia B
Intravenous infusion