According to a story from Angioedema News, the UK’s National Institute for Health and Clinical Excellence (NICE) has officially recommended the drug lanadelumab (marketed as Takhzyro) for coverage under the NHS as a treatment for swelling attacks associated with the rare disease hereditary angioedema (HAE). However, the organization has specified that the drug should be utilized only under certain conditions.
About Hereditary Angioedema
Hereditary angioedema is a genetic disorder which is characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations affecting the HAE gene. Swelling attacks generally occur every two weeks or so; they can usually last for several days. Swelling may affect the limbs, digestive tract, face, and airway, with blockage of the airway being the most dangerous complication. Vomiting and abdominal pain may accompany attacks as well if the digestive tract is involved. Treatment involves reducing the likelihood for attacks to appear and preventing them from worsening when they do. Hereditary angioedema is typically only life-threatening if left untreated. Prevalence of the condition is estimated to be around one in 10,000 to one in 50,000, at least in the US and Canada. To learn more about hereditary angioedema, click here.
NICE’s decision lists certain specifications regarding the use of Takhzyro:
- The drug should only be used in patients aged 12 or older.
- The developer continues to provide the medication under current commercial arrangements.
- Patients must also be eligible for prophylactic treatment with C1-inhibitors.
- Administration occurs at the ‘lowest dosing frequency’ compatible with the drug’s characteristics.
Within three months of the publishing of NICE’s Final Appraisal Determination (FAD), which is expected next month, the drug will become available on England’s NHS. The decision means that hereditary angioedema patients in the UK will have an all-new treatment option that will give them a greater opportunity to improve their quality of life and prevent dangerous swelling episodes.
Takhzyro has been approved in a number of developed countries around the world for the prevention of swelling attacks, including in the US, Australia, Switzerland, the EU, and Canada. It is the first preventative medication for the condition that can be self administered by patients.