TAKHZYRO® (lanadelumab injection) receives positive recommendation from CADTH's Canadian Drug Expert Committee (CDEC) for the routine prevention of hereditary angioedema (HAE) attacks

Saturday, November 23, 2019 Drug News
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The positive recommendation is supported by the recently released findings of the Phase 3 Help Study™ Open-Label Extension in HAE1

Patients living with HAE are now one step closer to having access to a first-of-its-kind monoclonal antibody treatment2

TORONTO, Nov. 22, 2019 /CNW/ - Takeda Canada Inc. is pleased to announce that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued a positive recommendation that TAKHZYRO® (lanadelumab injection) be reimbursed by public drug plans for routine prevention of hereditary angioedema (HAE) attacks in adolescents and adults (12 years of age and older).

"HAE is a chronic disease that results in acute attacks of swelling of various body parts and which can be life-threatening. As a physician who treats patients with HAE, I am pleased to have access to a treatment like TAKHZYRO to help prevent HAE attacks," said Stephen D Betschel, HBSc, MD, FRCPC, and Chair of The Canadian Hereditary Angioedema Network (CHAEN). "This is an exciting development for Canadian patients living with HAE, and with CADTH's positive recommendation, we are one step closer to getting access to this novel and important treatment option." 

Following a priority review, Health Canada authorized TAKHZYRO for routine prevention of attacks of HAE in adolescents and adults (12 years of age and older) in September 2018. Health Canada's authorization was based on findings from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™.3 This data is now further supported by the Phase 3 HELP Study™ Open-label Extension (OLE)i, that investigates the long-term safety and efficacy of TAKHZYRO. 

Presented at the 2019 American College of Allergy, Asthma and Immunology (ACAAI) Annual Meeting on November 7-11, the HELP Study OLE4 showed that the safety profile of TAKHZYRO was consistent with the original findings from the HELP Study, with treatment-related treatment emergent adverse events (TEAEs) occurring in 50% of patients (n=212).5 In addition, TAKHZYRO 300 mg every 2 weeks reduced the rate of attacks, attacks requiring acute treatment and moderate to severe attacks (secondary efficacy endpoints). The mean attack rate was reduced by 87% overall compared with baseline (n=212). Similarly there was an overall reduction of 92.6% in the rate of attacks requiring acute treatment (n=212) and 83.6% in the rate of moderate/severe attacks versus baseline (n=212).6 An exploratory analysis showed that the overall maximum attack-free period lasted ?12 months in 58% of patients (n=209) and ?6 months in 78% of patients (n=209) following the first regular treatment dose.7

In the study, TEAEs occurred in ~95% of patients (n=212) and were mostly mild or moderate in severity. The TEAEs related to treatment that were reported in more than 5% of patients were injection site pain, injection site erythema and injection site bruising.8

"We are hopeful that CADTH's positive recommendation will provide Canadians living with HAE access to a new and useful therapy to help bridge the gap in care", said Jacquie Badiou, President, Hereditary Angioedema (HAE) Canada. "HAE not only affects patients physically but can have detrimental impacts on one's quality of life due to the debilitating and unpredictable nature of the disease which is why new options are important." 

The Phase 3 HELP Study found that patients achieved an improvement in quality of life as measured by angioedema quality of life (AE-QoL) questionnaire.9 Findings showed that patients reported a quality of life improvement of 80.8% for TAKHZYRO dosed at 300 mg every 2 weeks and 63.0% for TAKHZYRO dosed at 300 mg every 4 weeks, compared to 36.8% for the placebo arm.10

"At Takeda, we are committed to providing new and innovative options for patients and their families and we are delighted with CADTH's positive recommendation for TAKHZYRO," said Gamze Yüceland, General Manager, Takeda Canada. "We look forward to working with our partners to bring this important therapy to Canadians living with HAE as soon as possible."

About The HELP Study Open-label Extension The HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE) is an evaluation of the long-term efficacy and safety of TAKHZYRO in HAE patients of at least 12 years of age and older. Two hundred and twelve patients received treatment with TAKHZYRO at the start of the OLE Study (109 rollover patients originally evaluated in the HELP Study and who continued into the OLE, and 103 eligible patients who did not participate in the HELP study but who had experienced at least one attack in 12 weeks). Rollover patients received a dose of 300 mg of TAKHZYRO on Day 0 and then every two weeks after their first attack. Non-rollover patients were treated with one 300 mg dose every two weeks, beginning on Day 0. One hundred and ninety-three participants completed at least 12 months of treatment and 27 participants completed at least 24 months of treatment.11,12

About TAKHZYRO (lanadelumab injection) TAKHZYRO (lanadelumab injection) is indicated for routine prevention of attacks of hereditary angioedema (HAE) in adolescents and adults (12 years of age and older).13  

TAKHZYRO is not intended for acute treatment of HAE attacks. Patients and caregivers should continue to be prepared to treat attacks with acute HAE treatments when necessary.14

TAKHZYRO is a fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein activity and is indicated for the routine prevention of HAE attacks in patients 12 years and older. TAKHZYRO is formulated for subcutaneous administration every two weeks.15 A dosing interval of 300mg every four weeks may be considered if a patient is well controlled (attack free) for more than six months.16 TAKHZYRO is intended for self-administration or administration by a caregiver. The patient or caregiver should be trained by a healthcare professional.17

It is not known if TAKHZYRO is safe and effective in children under 12 years of age.18

TAKHZYRO is approved in the U.S. (August 2018), E.U. (November 2018), Australia (January 2019) and Switzerland (June 2019) and additional regulatory submissions are ongoing worldwide.  TAKHZYRO was previously marketed in Canada by Shire Pharma Canada ULC, which was acquired by Takeda in January 2019.

About Hereditary Angioedema HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.19,20 It is estimated that there are approximately 880 HAE patients in Canada.21 The condition results in recurring attacks of edema (swelling) in various parts of the body that can be debilitating and painful.22,23,24,25 Laryngeal attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation.26,27,28 Aside from the burden of the disease itself, patients can suffer anxiety, fatigue and depression between attacks.29

About Takeda Canada Inc.Takeda Canada Inc. is the Canadian marketing and sales organization 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), Rare Diseases and Neuroscience. 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 Canada is available at www.takeda.com/en-ca 

____________________________i Interim Results from the HELP OLE Study

References_______________________________1 Riedl MA, Cicardi M, Hao J, et al; for the HELP OLE Study Investigators. Long-Term Efficacy of Lanadelumab: Interim Results from the HELP Open-Label Study. ACAAI 2019. Abstract #P1592 Riedl MA, Cicardi M, Hao J, et al; for the HELP OLE Study Investigators. Long-Term Efficacy of Lanadelumab: Interim Results from the HELP Open-Label Study. ACAAI 2019. Abstract #P1593 TAKHZYRO Product Monograph; September 2018 4 Riedl MA, Cicardi M, Hao J, et al; for the HELP OLE Study Investigators. Long-Term Efficacy of Lanadelumab: Interim Results from the HELP Open-Label Study. ACAAI 2019. Abstract #P1595 Johnston DT, Banerji A, Nurse C, et al; for the HELP OLE Study Investigators. Long-Term Safety of Lanadelumab in Hereditary Angioedeima (HAE): Interim Results From the HELP OLE Study. ACAAI 2019. Abstract #158.6 Riedl MA, Cicardi M, Hao J, et al; for the HELP OLE Study Investigators. Long-Term Efficacy of Lanadelumab: Interim Results from the HELP Open-Label Study. ACAAI 2019. Abstract #P1597 Riedl MA, Cicardi M, Hao J, et al; for the HELP OLE Study Investigators. Long-Term Efficacy of Lanadelumab: Interim Results from the HELP Open-Label Study. ACAAI 2019. Abstract #P1598 Johnston DT, Banerji A, Nurse C, et al; for the HELP OLE Study Investigators. Long-Term Safety of Lanadelumab in Hereditary Angioedeima (HAE): Interim Results From the HELP OLE Study. ACAAI 2019. Abstract #158.9 TAKHZYRO Product Monograph; September 201810 TAKHZYRO Product Monograph; September 201811 Riedl MA, Cicardi M, Hao J, et al; for the HELP OLE Study Investigators. Long-Term Efficacy of Lanadelumab: Interim Results from the HELP Open-Label Study. ACAAI 2019. Abstract #P15912 Johnston DT, Banerji A, Nurse C, et al; for the HELP OLE Study Investigators. Long-Term Safety of Lanadelumab in Hereditary Angioedeima (HAE): Interim Results From the HELP OLE Study. ACAAI 2019. Abstract #158.13 TAKHZYRO Product Monograph; September 201814 TAKHZYRO Product Monograph; September 201815 TAKHZYRO Product Monograph; September 201816 TAKHZYRO Product Monograph; September 201817 TAKHZYRO Product Monograph; September 201818 TAKHZYRO Product Monograph; September 201819 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):147-157.20 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.21 HAE Canada. About HAE. Retrieved November 1, 2019, from https://www.haecanada.org/about-hae/ 22 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):147-157.23 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.24 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.25 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.26 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):147-157.27 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.28 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.29 Fouche AS, Saunders EFH, Craig T. Depression and anxiety in patients with hereditary angioedema. Ann Allergy   Asthma Immunol. 2014;112(4):371–375.

SOURCE Takeda Canada



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