The National Institute for Health and Clinical Excellence (NICE), the health technology appraisal body in England and Wales, has issued its Final Appraisal Determination (FAD) recommending Takhzyro (lanadelumab) subcutaneous injection as an option for preventing recurrent attacks of HAE in patients aged 12 years and older only if: (1) they are eligible for preventive C1-esterase inhibitor (C1-INH) treatment in line with NHS England’s commissioning policy, (2) the lowest dosing frequency of lanadelumab is used in line with the summary of product characteristics, and (3) the company provides lanadelumab according to the commercial arrangement.
Laura Szutowicz, CEO of HAE UK said, “The 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.”
Lanadelumab is the first preventative treatment for Type I/II HAE, self-administered by patients at home as a single subcutaneous injection every two to four weeks. In clinical studies, monthly attack rates were reduced by 87 percent relative to placebo, and patients experienced improvements including less fear and shame about unpredictable attacks, less impairment in their ability to work, socialise, and perform other physical activities and reduced fatigue during the day and a better night’s sleep.
Dr Sinisa Savic, Consultant in Clinical Immunology and Allergy, said, “the replacement of frequent intravenous injections with at-home subcutaneous administration every two to four weeks, and the chance of being attack free, means that lanadelumab has the potential to transform care for some patients. It represents a real step-change in the treatment and clinical management of patients who experience recurrent HAE attacks.”
Key evidence behind the NICE recommendation for Takhzyro was data from the HELP-03 (Hereditary Angioedema Long-term Prophylaxis) Study. In this 26-week study, with 125 people with HAE, Takhzyro reduced the mean number of monthly HAE attacks by 87 percent compared with placebo when administered at 300 mg every two weeks and 73 percent compared with placebo when administered at 300 mg every four weeks A prespecified, exploratory analysis showed that over the entire 26-week study, 44 percent of patients taking Takhzyro 300 mg every two weeks were attack-free vs 2 percent of patients taking placebo. A post-hoc sensitivity analysis showed that 77 percent of the patients receiving Takhzyro 300 mg every two weeks were attack-free during a steady-state vs 3 percent of patients on placebo. The HELP Study is the largest randomised, controlled clinical prevention study conducted to date in this rare disease.
The FAD is part of the final guidance to the NHS in England and Wales expected to be published in October 2019. Pending any appeals from key stakeholders, based on this positive recommendation, the NHS should make Takhzyro available in England within three months of this date.
“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,” said Jon Neal, Managing Director, UK and Ireland, Takeda UK Ltd.“Takeda is really proud to be able to bring this novel medication to those living with this extremely debilitating condition.”
Takhzyro is approved in the United States of America (August 2018), Canada (September 2018), the European Union (November 2018), Australia (January 2019), and Switzerland (June 2019) and additional regulatory submissions are ongoing worldwide.
(Source: Takeda UK Ltd.)