The National Institute for Health and Clinical Excellence (NICE) - a health technology appraisal body in England and Wales - has recommended the use of Takeda’s Takhzyro (lanadelumab) on the NHS.
A final appraisal document has been published indicating the subcutaneous injection as an option for preventing recurrent attacks of hereditary angioedema (HAE) in people aged 12 and older, meaning that eligible patients can now receive the innovative treatment.
Patients will, however, only receive the drug if they are eligible for preventive C1-esterase inhibitor (C1-INH) treatment in line with NHS England’s commissioning policy and the lowest dosing frequency of Takhzyro is used in line with the summary of product characteristics.
The recommendation was made based partly on data from the HELP-03 Study, in which Takhzyro reduced the mean number of monthly HAE attacks by 87% compared with placebo when administered at 300 mg every two weeks and 73% compared with placebo when administered at 300 mg every four weeks.
“HAE attacks are unpredictable, painful and debilitating” reminded Laura Szutowicz, chief executive officer, HAE UK.
“Today’s recommendation from NICE means that people living with HAE across England and Wales have another medication that gives them the chance to lead a full and active life. We hope the NHS and Healthcare Professionals will provide swift access to patients who could benefit from this new treatment.”
HAE is a rare, genetic disorder that results in recurring attacks of swelling in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. Attacks that obstruct the airways can cause asphyxiation (suffocation) and are potentially life-threatening.