Uncovering the secrets of Fidanako-Ella Parvowitz gene therapy: What kind of treatment is it?

Fidanacogene-Ella Parvowitz (fidanacogene elaparvovec) gene therapy is an innovative one-time treatment drug for B hemophilia. It uses genetic engineering technology to convert functional coagulation factor IX< The /span>(FIX) gene is introduced into the patient’s liver cells, allowing them to independently synthesize the FIX protein, thus reducing or eliminating bleeding events.

Adult patients 18 years and older with moderate to severe hemophilia B who are receiving coagulation factor IX (FIX) prophylaxis, or who have life-threatening bleeding events or recurrent severe spontaneous bleeding.

The highly active variant of the FIX gene (FIX-Pa dua) is delivered to liver cells, allowing patients to autonomously produce FIX protein and reduce dependence on external FIX infusion.

Based on results from the single-arm, open-label Phase IIIBENEGENE-2 trial, which included 45 participants.

Effectiveness: The annualized bleeding rate (ABR) was reduced by 71%. The annualized coagulation factor transfusion rate was reduced by 92%. Some patients develop an immune response to treatment-targeted hepatocytes that can be controlled with steroid therapy.

On 20244month20, the U.S. Food and Drug Administration (FDA) approvedfidanacogene elaparvovecis used to treat B hemophilia. European Medicines Agency (EMA) has received a Marketing Authorization Application (MAA) for this therapy. Hemophilia B is a

fidanacogene elaparvovecAchieves long-term expression of FIX protein through a single treatment, providing patients with a potentially curative solution that significantly improves their bleeding control and quality of life.

Reference materials:https://en.wikipedia.org/wiki/Fidanacogene_elaparvovec