The Canadian Drug Expert Committee (CDEC) has recommended that Takhzyro (lanadelumb) be reimbursed by public drug plans when used as a preventive treatment for hereditary angioedema (HAE) attacks in patients 12 and older.
“HAE is a chronic disease that results in acute attacks of swelling of various body parts and which can be life-threatening. As a physician who treats patients with HAE, I am pleased to have access to a treatment like Takhzyro to help prevent HAE attacks,” Stephen D. Betschel, MD, and chair of The Canadian Hereditary Angioedema Network, said in a press release.
“This is an exciting development for Canadian patients living with HAE, and with CADTH’s positive recommendation, we are one step closer to getting access to this novel and important treatment option,” Betschel said.
CDEC is an arm of the Canadian Agency for Drugs and Technologies in Health (CADTH), an independent, not-for-profit organization responsible for providing healthcare decision-makers with evidence to make informed decisions.
“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 of Takeda Canada. “We look forward to working with our partners to bring this important therapy to Canadians living with HAE as soon as possible.”
Takhzyro — developed by Shire, which is now part of Takeda — is a human antibody that works by inhibiting the activity of the enzyme kallikrein and preventing the overproduction of bradykinin, a peptide that normally regulates blood pressure and inflammation by dilating blood vessels, and, in HAE, leads to bouts of swelling.
After giving Takhzyro priority review, Health Canada approved the medication in September 2018 as a routine preventive treatment for HAE attacks in people 12 and older.
At the time, the decision was based on data from the HELP Phase 3 trial (NCT02586805), and now it has been further supported by results from the trial’s open-label extension study (NCT02741596). The new findings were recently presented in three posters (P156, P158, and P159) at the 2019 American College of Allergy, Asthma and Immunology Annual Meeting in Houston, Texas.
The original HELP study was designed to evaluate the safety and effectiveness of Takhzyro in preventing HAE attacks in 125 patients with HAE over a period of 26 weeks.
Trial findings showed that Takhzyro reduced the frequency of HAE attacks by 87% when administered by a subcutaneous (under the skin) injection at a dose of 300 mg every two weeks, and by 73% when administered at the same dose once a month, compared to a placebo.
New data from the trial’s open-label extension study, which have also been published in the Annals of Allergy, Asthma & Immunology, showed that Takhzyro maintained its safety and efficacy at reducing the frequency of HAE attacks on a long-term basis.
The open-label extension study enrolled 212 patients, including 109 who had previously completed HELP and 103 who did not participate in HELP, but had experienced at least one HAE attack in the previous three months.
Those who participated in HELP received Takhzyro at a dose of 300 mg on day 0 and then every two weeks after having their first attack, while those who did not participate in HELP started treatment with Takhzyro (300 mg dose every two weeks) right away on day 0.
New data showed that in the overall population, Takhzyro reduced the frequency of HAE attacks by 87%, compared to baseline. Treatment reduced the rate of attacks requiring acute medical treatment by 92.6%, and the rate of moderate and severe attacks by 83.6%.
An exploratory analysis also revealed that 78% of the patients remained free of HAE attacks for more than six months and 58% for more than one year after receiving their first dose of Takhzyro.
Treatment safety analyses were consistent with previous data from HELP. During the open-label extension study, approximately 95% of the participants experienced treatment-emergent adverse events (TEAEs), but most of them were only mild or moderate in severity. The most common TEAEs reported during the study included injection site pain, erythema (skin redness), and bruising.
No serious TEAEs or deaths were reported in the course of the study.
“While the original HELP Study data has given us a strong understanding of how Takhzyro can prevent HAE attacks, the results of these analyses provide encouraging insight into its potential safety and efficacy over a longer duration,” Marc A. Riedl, MD, investigator in the open-label extension study, professor, and clinical director of the U.S. Hereditary Angioedema Association Center at the University of California, San Diego, said in a press release.
“As HAE is a lifelong condition, the long-term efficacy and safety of prophylactic medications are important factors in developing individualized HAE management plans. The HELP Study [open-label extension] analyses are a positive step forward in understanding the long-term effects of Takhzyro, and we look forward to gathering and sharing additional data as the study progresses,” Riedl added.