About Hemophilia A: Meaning and Therapies

 

What is Hemophilia A?

Hemophilia A is a genetic bleeding disorder in which an individual lacks or has low levels of proteins named clotting factor VIII.

The mainstay treatment option has long been FVIII replacement therapy. Initially, FVIII replacement was accomplished by donated whole blood, subsequently by plasma and currently by recombinant human FVIII (rFVIII) replacement therapies, which revolutionized the treatment of Hemophilia A.

About 30% of severe hemophilia A patients develop neutralizing anti-FVIII alloantibodies (inhibitors), which render the FVIII replacement ineffective. The standard of care therapy for patients with inhibitors is to induce immune tolerance with high-dose, high-frequency FVIII and treatment with bypassing agents (e.g. recombinant activated factor VII such as NovoSeven, FEIBA).

As per DelveInsight estimation, the future of Hemophilia A Market treatment is continuing to trend toward extended half-life therapies as well as more novel approaches including siRNA, bi-specific antibodies and gene therapy If these therapies are ultimately successfully commercialized they have the potential to transform the current standard of care for hemophilia A patients.

Hemophilia A is a general term for a group of rare bleeding disorders caused by a congenital deficiency of certain clotting factors. The main form of hemophilia is hemophilia A; it is different from Hemophilia B. The main difference between them is that Hemophilia B is associated with deficiency in factor IX (9) while Hemophilia A is associated with deficiency of factor VIII. More than 80% of people with hemophilia have hemophilia A.

Hemophilia A is a life-long condition. Currently, there is no cure, but researchers are actively engaged in finding the cure through gene therapy. One hope is that by inserting a healthy version of the defective blood factor gene, a person with hemophilia will be able to produce reasonable amounts of a factor on their own.

Generally, Hemophilia A patients are provided with “On Demand” and “Prophylaxis” treatment. But Prophylaxis treatment option has gained importance in comparison to the on-demand treatment options.

Check It’s — Hemophilia A Market Report

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