Hemophilia
A preclinical mouse study showed hemostasis restoration, as well as use for active bleeding and internal bleeding prevention.
Fidanacogene elaparvovec gene therapy is a safe and effective treatment for patients with hemophilia B, research shows.
Approval was based on data from phase 3 studies in the ongoing ATLAS clinical development program.
The Clinical Practice Guidance document issued by the EHA, ISTH, EAHAD, and ESO addresses both hemophilia A and B.
A study cohort with severe disease over a median follow-up of 14 months had an annualized bleeding rate of zero.
This approval for patients aged 12 years and older was based on findings from the explorer7 phase III clinical trial.
A new study addresses the need for data on treatment outcomes in patients younger than 12 years of age.
Quality of life improvement is among the benefits from this agent in patients with congenital disease.
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