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Retracing the launch of Fidanaco-Ella Parvowitz gene therapy
Fidanaco-Ellaparvitz gene therapy was approved by the U.S. Food and Drug Administration (FDA) on April 26, 2024, for the treatment of adult patients 18 years of age and older with moderate to severe hemophilia B and who test negative for neutralizing antibodies to AAV serotype Rh74. This approval marks the formal entry of this therapy into clinical application, providing a new treatment option for patients with hemophilia B.
The development of this therapy began with in-depth exploration of the pathological mechanisms of hemophilia B. Researchers have discovered that hemophilia B is mainly caused by a deficiency of coagulation factor IX (FIX). Therefore, supplementing FIX through gene therapy has become the key to treating the disease.
Based on the positive results of the clinical trial, Pfizer submitted a Biologics License Application (BLA) to the FDA. After rigorous review and evaluation, the FDA approved the marketing application for this therapy on April 26, 2024.
The launch of Fidanaco-Ella Parvowitz gene therapy brings new treatment hope to patients with hemophilia B. The therapy, administered via a single intravenous infusion, achieves long-term effects and significantly reduces or eliminates patients' dependence on external coagulation factor infusions. At the same time, the safety of this therapy has also been verified, providing patients with a safer and more effective treatment option.
Reference: https://en.wikipedia.org/wiki/Fidanacogene_elaparvovec
The development of this therapy began with in-depth exploration of the pathological mechanisms of hemophilia B. Researchers have discovered that hemophilia B is mainly caused by a deficiency of coagulation factor IX (FIX). Therefore, supplementing FIX through gene therapy has become the key to treating the disease.
In the selection of gene vectors, the researchers used adeno-associated virus (AAV) as the vector because of its advantages of low immunogenicity and high transduction efficiency. At the same time, the AAV vector has been optimized to improve its targeting and safety.
Based on the positive results of the clinical trial, Pfizer submitted a Biologics License Application (BLA) to the FDA. After rigorous review and evaluation, the FDA approved the marketing application for this therapy on April 26, 2024.
The launch of Fidanaco-Ella Parvowitz gene therapy brings new treatment hope to patients with hemophilia B. The therapy, administered via a single intravenous infusion, achieves long-term effects and significantly reduces or eliminates patients' dependence on external coagulation factor infusions. At the same time, the safety of this therapy has also been verified, providing patients with a safer and more effective treatment option.
Reference: https://en.wikipedia.org/wiki/Fidanacogene_elaparvovec
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