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First! FDA approves gene therapy for adult hemophilia

 Last Update: 2023-01-05
 Source: Internet
 Author: User

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Today, the U.
S.
Food and Drug Administration (FDA) announced the approval of CSL Behring's gene therapy Hemgenix (etranacogene).
Dezaparvovec).

The same press release states that this is the first FDA-approved gene therapy to treat adult patients with hemophilia B!

The same press release states that this is the first FDA-approved gene therapy to treat adult patients with hemophilia B!

Hemophilia B is a life-threatening degenerative disease in which patients are deficient in clotting factor IX (FIX)
due to genetic mutations.
Patients with the disease are prone to bleeding in joints, muscles, and internal organs, with pain, swelling, and joint damage
.
Current treatment includes lifelong prophylactic transfusion of clotting factor IX to temporarily replace or replace low levels of clotting factors
.

Hemophilia B is a life-threatening degenerative disease in which patients suffer from a genetic mutation that results in a deficiency of clotting factor IX.
(FIX)

Today's approved Hemgenix uses AAV5 vectors to deliver coagulation factor IX Transgenic of the Padua variant, which is 5-8 times more active than ordinary coagulation factor IX, can complete normal coagulation function
at lower expression levels.
The FDA press release states that the therapy delivers genes to the liver, and the newly synthesized clotting factor IX can promote blood clotting and reduce bleeding events
.
Previously, the therapy had been granted breakthrough therapy designation, orphan drug designation, and priority review status by the US Food and Drug (FDA
).

The approval is based on the results of
two clinical trials.
These trials enrolled 57 patients with severe or moderately severe hemophilia B, and one trial showed that the therapy significantly increased the level of clotting factor IX in patients and reduced prophylactic treatment
of factor IX.
Another study showed that the treatment reduced annual bleeding rates
by 54%.

One trial showed that this therapy significantly increased the level of coagulation factor IX in patients and reduced the prophylactic treatment
of coagulation factor IX.
Another study showed that the treatment reduced annual bleeding rates
by 54%.

It is worth mentioning that previous clinical trials have also evaluated the impact of a patient's own immune system on gene therapy for the first time – the results showed that no evidence of
therapeutic activity was found in patients with AAV-neutralizing antibody titers as high as 678.
2.

The most common adverse effects of this gene therapy are elevated liver enzyme levels, headaches, mild transfusion-related reactions, and flu-like symptoms
.
The FDA also notes that doctors need to test patients for adverse reactions
in their blood.

"Today's approval provides a completely new treatment option for patients with hemophilia B and represents an important advance
in the development of innovative therapies.
" Peter of the FDA's Center for Biologics Evaluation and Research Dr.
Marks said
.

Today's approval provides a completely new treatment option for patients with hemophilia B and represents an important advance
in the development of innovative therapies.

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