Real world study in Canada shows Takhzyro cuts HAE attacks by 80%
Findings support injection therapy's use for long-term prevention
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Using Takhzyro (lanadelumab) cut monthly swelling attacks by about 80% in newly treated people with hereditary angioedema (HAE) in Canada, and helped maintain low attack rates among those already on the therapy.
Further, the injection treatment was generally well tolerated, with no serious side effects reported.
These findings come from an analysis of outcomes among Canadian participants with HAE enrolled in the Phase 4 EMPOWER study (NCT03845400), which spanned 29 locations in the U.S., Canada, and Puerto Rico. The study evaluated the long-term effectiveness and safety of Takhzyro in real-world settings among more than 100 people with HAE.
While the number of Canadians in the study was small, the researchers said the results nonetheless support “the use of [Takhzyro] as a first-line long-term [preventive] treatment for patients with HAE in Canada.”
Their report, titled “Long-term effectiveness and safety of lanadelumab in Canadian patients with hereditary angioedema: a subanalysis of the EMPOWER study,” was published in the journal Allergy, Asthma & Clinical Immunology. The study was sponsored by Takeda, the company that markets the therapy.
A rare genetic disorder, HAE is characterized by recurrent episodes of swelling that can affect the abdomen, face, hands, airway, and other parts of the body. These attacks can be painful and may impair daily life activities.
Little real-world data on Takhzyro use in Canada
Takhzyro is approved in the U.S., Canada, and other regions for the prevention of swelling attacks in children and adults with HAE. International and Canadian guidelines recommend its use as a first-line preventive option. The therapy, given as an injection under the skin (subcutaneously), is designed to block kallikrein, thereby reducing levels of bradykinin, a protein whose overactivity drives HAE swelling attacks.
“However, partly due to the rarity of the condition in the country, little information is available on outcomes from its use in real-world clinical practice in Canadian patients,” the researchers wrote.
To address this gap, the team analyzed outcomes in 13 people with HAE in Canada who enrolled in the EMPOWER study between November 2019 and December 2021. Seven were newly starting Takhzyro or had received fewer than four doses at enrollment. The remaining six had received at least four doses and were either still on Takhzyro or had received their last dose within 70 days before joining the study.
Participants were followed for up to 24 months, or about two years, unless they died, were lost to follow-up, or withdrew earlier.
Most of the patients were women (69%), and the mean age was 45.6 years. Nearly all (92%) had HAE type 1, the most common form of the disease. Altogether, 69% reported a family history of the disease.
Among individuals newly starting Takhzyro, the mean treatment duration was 315 days, or about 10 months. For those already on the therapy, the mean duration was 536 days, or approximately 1.5 years.
Low attack rates seen across all patient groups
Treatment with Takhzyro was linked to low attack rates in both groups, the analysis found. Patients already on Takhzyro maintained a mean of 0.1 attacks per month throughout the study and experienced seven attacks in total. All were mild or moderate, and lasted a mean of 26.4 hours, or slightly longer than one day. All but one of the patients were treated with on-demand medication, the data showed.
Newly treated patients saw an 80% drop in monthly attack rates, from a mean of 1.5 before treatment to 0.3 during follow-up. These participants experienced 31 attacks overall, lasting a mean of 17.5 hours, with 93.5% classified as mild or moderate. All were treated with on-demand medication.
No patients in either group required emergency room care for an attack.
Four patients were able to extend their dosing interval from every two weeks to every four weeks during the study. Among them, one was in the newly treated group and three were already on therapy.
Results from the subset of Canadian patients in the EMPOWER study support the use of [Takhzyro] as a first-line option for [long-term preventive treatment] in Canadian patients with HAE.
The treatment was generally well tolerated. No injection-site reactions were reported and no participants discontinued due to side effects. The most common side effects were mild or moderate, and no new safety concerns arose relative to previous studies.
“Results from the subset of Canadian patients in the EMPOWER study support the use of [Takhzyro] as a first-line option for [long-term preventive treatment] in Canadian patients with HAE,” the researchers concluded.
The team noted, however, that the small number of participants may limit how broadly the findings apply to the wider Canadian population.
Still, this analysis contributes “important information to the growing body of literature on [long-term preventive treatment] use among patients with HAE in Canada,” the team wrote.
