The UK’s National Institute for Health and Clinical Excellence (NICE) has recommended the use of subcutaneous injection of Takeda’s Takhzyro (lanadelumab) to prevent recurrent attacks of hereditary angioedema (HAE).
In its final appraisal determination (FAD), NICE recommended the treatment as an option for patients aged 12 years and above who are candidates for preventive C1-esterase inhibitor (C1-INH) treatment.
Takhzyro will be supplied under a commercial agreement by the company and can be used only in the lowest dosing frequency.
HAE is a rare, genetic disease that causes recurrent oedema attacks in different body parts, such as the face, abdomen, feet, genitals, hands and throat. Attacks can last for several days, with varying frequency.
Lanadelumab is a fully human monoclonal antibody that minimises plasma kallikrein activity. It is produced in Chinese Hamster Ovary (CHO) cells using recombinant DNA technology.
The drug secured the European Medicines Agency (EMA) approval in November last year as a preventive therapy for recurrent HAE attacks. The drug has been designed for at-home administration and can be taken every two to four weeks.
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Takeda noted that lanadelumab reduced monthly attack rates by 87% in clinical studies that compared it to placebo. Also, patients are said to have experienced improvements in quality of life.
The NICE recommendation comes after a review of data from the 26-week, 125-patient HELP-03 study, where the drug led to 87% and 73% decrease in the mean number of monthly HAE attacks when a 300mg dose was given every two weeks and four weeks, respectively.
An exploratory analysis revealed that 44% of participants on Takhzyro every two weeks were attack-free over the complete study duration compared to 2% with placebo.
According to findings from a post-hoc sensitivity analysis, 77% of subjects treated with the drug every two weeks were attack-free during a steady-state versus 3% of those on placebo.
Takeda UK and Ireland managing director Jon Neal said: “Ensuring people living with rare diseases, such as HAE, have the best care and access to innovative treatments is of the utmost importance to us and this recommendation by NICE represents a huge milestone.”
As well as the European Union (EU), Takhzyro has approvals in the US, Canada, Australia and Switzerland.