Haegarda Now Available to HAE Patients Across Canada

Haegarda is now available to adolescent and adult hereditary angioedema (HAE) patients across Canada for the prevention of HAE attacks, according to CSL Behring, the therapy’s developer.

The company announced its preventive treatment, made available for eligible HAE patients in April, can now be given in all of Canada without any restrictions.

It first became available to patients in the Canadian province of Quebec in February 2020.

Following this announcement, HAE Canada, a Canadian patient advocacy organization, issued a statement saying its president and board of directors “are excited that at long last, Haegarda is now equally available, without restrictions, to adolescent and adult HAE patients across Canada.”

“This represents yet another milestone for the advancement of care for Canadian HAE patients,” the organization said.

Haegarda works by replacing C1-inhibitor (C1-INH), a blood enzyme that is either missing or malfunctioning in HAE. The replacement therapy is designed to prevent the sudden and recurrent swelling attacks that occur in HAE patients due to the lack of the enzyme.

The medication is given as an under-the-skin (subcutaneous) injection, allowing for patients to administer it themselves. That’s a clear advantage compared with other replacement therapies that are given intravenously, or into the bloodstream, and are usually administered in a clinic or doctor’s office.

“As a physician that treats this disease, I’m pleased to have Haegarda available for patients across Canada,” said Stephen D. Betschel, MD, a clinical immunologist and allergist at St. Michael’s Hospital, in Toronto.

“Haegarda represents another needed therapeutic  advancement for the prevention of HAE as the only [under-the-skin] C1 Inhibitor for preventative therapy approved in Canada,” he said.

The therapy’s approval in Canada was supported by data from the Phase 3 COMPACT trial (NCT01912456), which was completed in 2015. That trial assessed Haegarda’s safety and effectiveness — when administered twice-weekly — compared with a placebo, in 90 patients, ages 12 and older, with type 1 or 2 HAE.

Results showed that Haegarda lowered the frequency of HAE attacks by a median of 95% compared with a placebo when given at its currently approved dose of 60 international units per kilogram (IU/kg), twice weekly.

The need for rescue therapies also was reduced by more than 99%. Additional analyses demonstrated the therapeutic benefits of Haegarda were evident within the first two weeks of treatment, during which HAE attacks were effectively reduced regardless of patient characteristics at study entry.

Through its CSL Behring PLUS+ program, the company also is providing patients with training on how to self-administer Haegarda, as well as additional educational materials. Those who are interested in joining the program must contact their healthcare providers.