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 HAE attacks in adolescents and adults (12 years of age and older).
“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 to 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™. This data is now further supported by the Phase 3 HELP Study™ Open-label Extension (OLE), 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 OLE 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). 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). 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.
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.
“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. 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.
“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 Inc. “We look forward to working with our partners to bring this important therapy to Canadians living with HAE as soon as possible.”