Study: Phase 3 Trial of TAKHZYRO, a Hereditary Angioedema Treatment

According to an article from Biospace, Takeda Pharmaceuticals has released the results from their Phase 3 HELP trial, an extension study of TAKHZYRO. This hereditary angioedema (HAE) treatment has shown to lower the frequency of attacks and even prevent them altogether.

About HAE

Hereditary angioedema (HAE) is a rare, genetic condition that is characterized by episodes of severe swelling of the blood vessels. While these attacks can be triggered by things like stress or injuries, they often occur without cause. Regardless of reason, symptoms include swelling of the extremities, facial swelling to the point where the eyes swell shut, severe abdominal swelling that can cause vomiting, nausea, and pain, and swelling in the throat that can make it difficult or impossible to breathe. These symptoms are the result of a genetic mutation on chromosome 11 that are inherited in an autosomal dominant pattern. When this mutation appears, a protein called C-1 inhibitor, is unable to properly do its job. This leads to an excess of a peptide called bradykinin, which causes inflammation throughout the body.

About the Trial

Researchers at Takeda have found that TAKHZYRO is a viable treatment option for HAE patients over the age of 12, as it is able to reduce the frequency of attacks and even prevent them. The safety profile remained consistent with previous research as well. Results include:

  • The mean rate of attacks were decreased by 87.4%
  • Almost 70% of participants experienced 12 months without an attack
  • 54.7% of participants experienced at adverse event, with the most common being injection-site pain, headache, or infections in the respiratory tract
  • 93% of patients experienced at least a 70% reduction in attacks

As an HAE attack can be severe and even life-threatening, it is absolutely necessary to have viable treatments options. Hopefully TAKHZYRO continues to see success in its development and is able to reach patients.

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