Pharma Global News: FDA approves first gene therapy for Hemophilia B

Pharma Global News: U.S. Food and Drug Administration (FDA) has approved Hemgenix (etranacogene dezaparvovec), the first gene therapy for adults with Hemophilia B.

Particularly, this gene therapy is used in adults who currently use Factor IX prophylaxis therapy, or those who have a current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.

Therefore, replacement of the lacking or insufficient clotting factor is the standard course of treatment for the condition to increase the body’s capacity to stop bleeding and encourage healing.

Similarly, to maintain adequate clotting factor levels to prevent bleeding episodes, patients with severe hemophilia B often require routine intravenous (IV) infusions of Factor IX replacement medicines.

Hemgenix gene therapy for Haemophilia B

Adeno-associated virus-based IV infusion Hemgenix carries the clotting Factor IX gene.

Therefore, to enhance blood levels of Factor IX and prevent bleeding episodes, the gene is released in the liver, where it produces the Factor IX protein.

Although, Hemgenix gene therapy indicates significant advancement in the development of novel therapeutics for this type of hemophilia and is a new therapy option.

Hemophilia B Clinical Trial

A study was carried out on 57 adult males in the age range of 18-75 years diagnosed with severe or moderately severe Hemophilia B.

Significantly, reductions in the men’s annualized bleeding rate (ABR) were used to measure the effectiveness 

Therefore, the results indicated increased Factor IX activity levels, reduced demand for routine Factor IX replacement prophylaxis, and a 54% drop in ABR in comparison to baseline.

Though, Hemgenix is approved it is still under assessment by regulatory agencies

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