New First-In-Class Drug for Hemophilia A Approved
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New First-In-Class Drug for Hemophilia A Approved.
On February 23 , the U.S. Food and Drug Administration ( FDA ) approved Sanofi’s Altuviiiio ( efanescoctcog alfa ) for routine prophylaxis and on-demand treatment in adult and pediatric patients with hemophilia A to control bleeding episodes and perioperatively period management.
Altuviiiio, jointly developed by Sanofi and Sobi , is the first FDA- approved First-In-Class high-sustainability factor VIII replacement therapy. Altuviiiio is based on innovative Fc fusion technology with the addition of von Willebrand factor and XTEN® polypeptide domains to extend its circulation time half-life, which is 3 to 4 times longer than standard and extended half-life Factor VIII products .
Therefore, the drug only needs to be taken once a week, can provide normal to near normal factor activity levels (over 40% ) most of the week , and can significantly reduce bleeding compared with previous factor VIII prophylaxis.
The FDA approval is based on data from the pivotal Phase III study of XTEND-1 recently published in the New England Journal of Medicine .
Once-weekly prophylaxis with Altuviiiio met its primary endpoint, providing significant bleeding protection in patients with severe hemophilia A , with a mean annual bleeding rate ( ABR ) of 0.70 ( 95% CI : 0.5-1.0 ) and a median ABR of 0.0 ( Q1 , Q3 : 0.0 , 1.0 ).
Altuviiiio met the key secondary endpoint, showing a significant 77% reduction in ABR ( 95% CI: 58%-87% ) compared to prior factor prophylaxis based on within-patient comparisons.
Hemophilia A is a rare, lifelong disease that causes a person’s blood to fail to clot properly, leading to external bleeding, bruising, and joint bleeding.
Bleeding in the joints can lead to loss of mobility and chronic pain, which is why Altuviiiio ‘s long-term efficacy is critical, Sanofi’s chief medical officer and head of global development Dietmar Berger , MD, Ph.D., said in a recent interview. The hemophilia A market is worth more than $ 10 billion , Berger said .
The FDA granted Altuviiio Breakthrough Therapy Designation in May 2022 , Fast Track Designation in February 2021 , and Orphan Drug Designation in August 2017 .
It is expected to submit a marketing application in the EU in the second half of 2023 . The European Commission granted the drug Orphan Drug designation in June 2019
New First-In-Class Drug for Hemophilia A Approved
(source:internet, reference only)
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