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NICE Recommends Takhzyro®▼(Lanadelumab) for the Treatment of People Living with Rare Debilitating Genetic Disorder

−  Takeda UK Ltd. is pleased to announce that NICE has recommended the use of Takhzyro®(lanadelumab) subcutaneous injection as an option for preventing recurrent attacks of hereditary angioedema (HAE) in people aged 12 and older, within their final appraisal document (FAD) published today[i]

−  This positive recommendation from NICE means that eligible patients can now receive this innovative subcutaneous treatment

−  HAE is a rare genetic disorder that can result in unpredictable and potentially life-threatening, recurring attacks of debilitating and painful swelling in various parts of the body which can last for a number of days [ii] [iii] [iv] 

HIGH WYCOMBE, England, Sept. 18, 2019 /PRNewswire/ -- Takeda UK Ltd. is pleased to announce that today 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 hereditary angioedema (HAE) in patients aged 12 years and older only if:

(PRNewsfoto/Takeda UK Ltd.)
  • they are eligible for preventive C1-esterase inhibitor (C1-INH) treatment in line with NHS England's commissioning policy
  • the lowest dosing frequency of lanadelumab is used in line with the summary of product characteristics
  • the company provides lanadelumab according to the commercial arrangement.i  

HAE is a rare, genetic disorder that results in recurring attacks of oedema (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.ii iii iv Attacks can last for a number of days,iii and the frequency may vary with some people having attacks on average every 1 to 2 weeks, or some experiencing an attack every three days.iv [v] Anxiety about the next attack can limit the way people with HAE live their life, for example avoiding educational and career opportunities, planning holidays and attending social engagements.[vi] 

Laura Szutowicz, Chief Executive Officer, HAE UK said, "HAE attacks are unpredictable, painful and debilitating. 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."

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.[vii]  In clinical studies, monthly attack rates were reduced by 87% 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. vii

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 the 26-week Phase 3 HELP Study™, with 125 people with HAE, 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 (adjusted P>0.001). A prespecified, exploratory analysis showed that over the entire 26-week study (Days 0-182), 44% (n=12/27) of patients taking Takhzyro 300 mg every two weeks were attack-free vs. 2% (n=1/41) of patients taking placebo. A post-hoc sensitivity analysis showed that 77% (n=20/26) of the patients receiving Takhzyro 300 mg every two weeks were attack-free during a steady-state (day 70-182) vs. 3% of patients on placebo (n=1/37). The HELP Study is the largest randomised, controlled clinical prevention study conducted to date in this rare disease.vii 

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 upmost importance to us and this recommendation by NICE represents a huge milestone," said Jon Neal, Managing Director, UK and Ireland, Takeda. "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 U.S. (August 2018), Canada (September 2018), E.U. (November 2018), Australia (January 2019) and Switzerland (June 2019) and additional regulatory submissions are ongoing worldwide.

About Takhzyro (lanadelumab)

Takhzyro is indicated for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients 12 years or older.  Takhzyro is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity. Takhzyro is produced in Chinese Hamster Ovary (CHO) cells by recombinant DNA technology. Takhzyro is approved by the European Medicines Agency (EMA) for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients 12 years and older. Takhzyro is formulated for subcutaneous administration and has a half-life of approximately two weeks in patients with HAE. Takhzyro is intended for self-administration or administration by a caregiver, only after training by a healthcare professional.vii

Please refer to lanadelumab EU Summary of Product Characteristics (SmPC) for more information.

https://www.medicines.org.uk/emc/product/9778

About The HELPStudy 

The HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ was a multicentre, randomised, double-blind, placebo-controlled parallel group trial that evaluated the efficacy and safety of subcutaneously administered Takhzyro vs. placebo over 26 weeks in 125 patients 12 years of age or older with HAE. The primary endpoint of the HELP Study™ was the number of investigator-confirmed HAE attacks over the entire 26-week study duration. Takhzyro demonstrated that subcutaneous injections every two or four weeks reduced the mean monthly number of attacks across all three Takhzyro treatment arms studied: 300 mg every two weeks, 300 mg every four weeks, and 150 mg of Takhzyro every four weeks. Complete results from the Phase 3 HELP Study™ were published in the Journal of the American Medical Association (JAMA) on 27 November 2018.[viii]

About Hereditary Angioedema (HAE)

HAE is a rare genetic disorder that results in recurring attacks of oedema—swelling—in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful. Attacks that obstruct the airways can cause asphyxiation and are potentially life-threatening.ii iii iv In the UK, HAE affects approximately 1 in 50,000 of the population[ix] and an estimated 1 in 10,000 to 1 in 50,000 people worldwide.iii [x]

About Takeda UK Ltd.

Takeda UK Ltd., located in High Wycombe, is the UK marketing and sales organisation of Takeda Pharmaceutical Company Limited, headquartered in Japan. Takeda Pharmaceutical Company Limited (TSE: 4502, NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Neuroscience and Rare Diseases. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

Additional information about Takeda UK Ltd. is available through its corporate website, www.takeda.com/en-gb.

References

[i] NICE. https://www.nice.org.uk/guidance/indevelopment/gid-ta10333. Last accessed: September 2019.

[ii] Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.

[iii] Cicardi M, Bork K, Caballero T, et al, on behalf of HAWK (Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012;67(2):147-157.

[iv] Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

[v] Bork K, Meng G, Staubach P, Hardt J. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med 2006;119:267-274

[vi] Bygum A et al. Burden of illness in hereditary angioedema: a conceptual model. Acta Derm Venereol 2015; 95:706–710.

[vii] Takhzyro® (lanadelumab) Summary of Product Characteristics (SmPC).

[viii] Banerji A et al. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks: a randomized clinical trial. JAMA 2018; 320:2108–2121.

[ix] British Society for Immunology. Hereditary angioedema.https://www.immunology.org/public-information/bitesized-immunology/immune-dysfunction/hereditary-angioedema. Last accessed: September 2019.

[x] Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.

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