The availability announcement was issued by Canadian Blood Services (CBS), the country’s blood authority, which is responsible for managing the blood supply in all provinces and territories, except Quebec. The therapy has been available in that city since February 2020.
“This accomplishment did not happen overnight; there were many steps taken to reach this amazing point in the road,” HAE Canada, a patient advocacy group that was involved in the process by providing a patient submission to CBS, stated in a press release.
Besides its members and CSL Behring, HAE Canada also thanked CBS “for listening to patients who shared their stories and experiences and for ultimately adding this treatment to the CBS formulary, allowing equitable access to patients across Canada.”
Haegarda works by providing patients the C1-inhibitor (C1-INH), the enzyme that is either missing or malfunctioning in HAE. As such, the therapy is expected to prevent the sudden and recurrent swelling attacks caused by the lack of C1-INH.
Unlike other replacement therapies that are administered directly into the bloodstream through into-the-vein infusions, Haegarda is given as an under-the-skin injection, allowing for self-administration.
Its approval was based on data from the Phase 3 COMPACT clinical trial (NCT01912456), which evaluated the safety and effectiveness of a twice-weekly injection of Haegarda, compared with a placebo, in 90 patients, ages 12 and older, with type 1 or 2 HAE.
The results showed that Haegarda, when given at the highest and currently approved dose (60 international units/kg), safely and substantially lowered the frequency of swelling attacks by 95%, compared with a placebo. The therapy also reduced the need for rescue therapies by more than 99%.
Further analysis also highlighted that Haegarda’s therapeutic benefits were evident within two weeks of the treatment’s start, and that treatment effectively reduced HAE attacks regardless of patient characteristics at study entry.
CSL Behring Canada has an assistance program, called Haegarda Connect, to aid patients in learning the best method of self-administering Haegarda, as well as having access to personalized support and financial assistance programs. Interested patients must ask their physicians to join the program.
In the U.S., the therapy’s original label, for patients ages 12 and older, was extended last year to include children, ages 6 and older.
In a separate release, the Canadian patient advocacy organization also announced that Takeda’s Takhzyro (lanadelumab), another preventive HAE treatment, became available at low or no cost to eligible patients in Newfoundland and Labrador on March 17.
Eligible patients include those ages 12 and older who have experienced at least three HAE attacks within a one-month period and needed an injectable treatment.
Similar decisions have already occurred in other provinces, such as Alberta, Quebec, Ontario, British Columbia, New Brunswick, and Manitoba.
The therapy was approved in Canada in 2018. But only in November 2020 was Takeda Canada able to reach an agreement with the pan-Canadian Pharmaceutical Alliance for Takhzyro’s public funding. Each Canadian provincial and territorial government then decides whether or not to provide the therapy to patients.
Developed by Shire, which is now part of Takeda, Takhzyro is a human antibody that works by blocking the activity of plasma kallikrein — a precursor of bradykinin, the inflammatory molecule produced in excess in HAE patients that leads to swelling attacks.
Its approval was based on data from the Phase 3 HELP trial (NCT02586805), which showed that Takhzyro, given at 300 mg every two weeks, led to an 87% drop in the mean number of HAE attacks per month, compared with a placebo. The trial enrolled 125 patients, ages 12 and older, with type 1 or 2 HAE.
The therapy also lowered the number of attacks requiring acute treatment and the rate of moderate to severe attacks.
Takhzyro also has been approved to prevent swelling attacks in adolescents and adults with HAE in the U.S., the European Union, Australia, Switzerland, and Brazil, among other countries.
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