Shire plc and Shire Pharma Canada ULC (Shire Canada) announces that following priority review, Health Canada has authorized TAKHZYRO (lanadelumab injection) for routine prevention of attacks of HAE in adolescents and adults (12 years of age and older).
“The burden HAE patients and their families face every day can’t be ignored,” said Jacquie Badiou, President, HAE Canada. “Our National Report Card shows that the unpredictable nature of this life-threatening disorder, not knowing when the next attack will come, has a negative impact on their lives and the lives of their loved ones. This new treatment option is great news as it was shown to significantly reduce the frequency of attacks.”
TAKHZYRO is a fully human monoclonal antibody that inhibits the activity of plasma kallikrein, an enzyme which is uncontrolled in people with HAE, to prevent attacks. The recommended dose of TAKHZYRO is 300 mg every 2 weeks. A dosing interval of 300 mg every 4 weeks may be considered if the patient is well-controlled (e.g., attack free) for more than 6 months.
Health Canada’s authorization of TAKHZYRO for the routine prevention of HAE attacks in adolescents and adults is supported by results of the Phase III HELP (Hereditary Angioedema Long-term Prophylaxis) Study, in which the primary efficacy endpoint was the number of investigator-confirmed HAE attacks during the 26-week study duration. The HELP study demonstrated that TAKHZYRO reduced the number of monthly HAE attacks by an average of 87 percent (n=27) vs. placebo (n=41) when administered at 300 mg every two weeks and 73 per cent (n=29) vs. placebo (n=41) when administered at 300 mg every four weeks (P<0.001).
In the 26-week clinical study, which enrolled 125 patients with HAE with CI inhibitor deficiency, secondary endpoints included: the number of attacks requiring acute treatment and the number of attacks assessed as moderate or severe. Patients taking TAKHZYRO 300 mg every two weeks had 87 percent fewer attacks that required on-demand treatment and 83 percent fewer moderate to severe attacks. A pre-specified, exploratory analysis showed that 44 percent of patients (n=27) receiving TAKHZYRO 300 mg every two weeks had zero attacks compared to placebo (2 percent, n=41) for the 26-week treatment period.
Of the patients who received TAKHZYRO and completed the HELP Study, the largest prevention study conducted to date in HAE, 97 percent enrolled in an ongoing open-label extension study designed to evaluate the long-term safety and efficacy of TAKHZYRO. In the extension study, after receiving a single dose of TAKHZYRO 300 mg at study entry, 80 percent of patients who had been in the 300 mg every two weeks treatment group (n=25) in the HELP Study remained attack-free at week 4 post-dose.
The most common side effects seen with TAKHZYRO were injection site reactions including pain, redness, and bruising.
“As a physician who treats patients with HAE, I am pleased to have a treatment like TAKHZYRO available for the prevention of HAE attacks,” said Dr. Stephen D. Betschel. “The HAE community has a new option that can help prevent attacks.”
TAKHZYRO has a half-life of approximately 14 days and can be self-administered every two or four weeks as one subcutaneous injection. In the HELP Study extension the majority of self-injections took one minute or less to complete.
“This authorization reinforces our ongoing commitment to develop innovative therapies that can help make a positive impact on patients,” said Eric Tse, General Manager, Shire Canada. “We will continue to work towards our goal of improving the lives of those living with rare diseases, including HAE.”