The US Food and Drug Administration has approved a drug called TakhzyroTM (lanadelumab-flyo) for the treatment of hereditary angioedema. This approval was based on supporting evidence from several studies, including from a HELPTM clinical trial. For more detailed information you can view the source press release here, at Globe Newswire.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a genetic condition that is linked to periods of swelling of various areas that typically last a few days. These attacks can affect areas such as the throat, face, hands, and intestines, amongst others. Swelling affecting the intestinal wall may cause people with HAE to experience excruciating abdominal pain, nausea, and vomiting. According to the US Hereditary Angioedema Association, HAE affects an estimated one in 10,000 to 50,000 people.
About TakhzyroTM (lanadelumab-flyo)
Takhzyro is a monoclonal antibody that works to prevent attacks by inhibiting an enzyme called plasma kallikrein that is chronically uncontrolled in people who have HAE. Takhzyro has recently been approved by the FDA for treatment of HAE in patients aged 12 years and above, under the supervision of a medical professional.
The HELPTM Clinical Trial
The HELP (Hereditary Angioedema Long-term Prophylaxis) study was a Phase 3 clinical trial that compared the effects of Takhzyro to a placebo. 125 people with HAE took part in the 26 week study. Data from the study showed that Takhzyro was linked to an 87% reduction in monthly HAE attacks when given every two weeks, and by 73% when given every four weeks. Furthermore, the study met all its secondary endpoints, including a reduction in the number of moderate or severe attacks and attacks that required acute treatment.
97% of the patients who completed this study and received Takhzyro chose to take part in an on-going open-label extension study that will investigate the long-term safety and effectiveness of the drug.
For more information about the FDA’s decision, and the studies underlying it, you can click here to view the original press release.