Takhzyro effective in preventing HAE attacks: Real-world study
Median of 2.2 monthly attacks falls to zero with treatment, clinic data show
Takhzyro (lanadelumab), an injectable medication used to prevent hereditary angioedema (HAE) attacks, is highly effective at reducing the frequency of these episodes and increasing the proportion of patients who remain attack-free, a study into its real-world use reports.
Researchers evaluated Takhzyro’s use in the clinic among HAE patients in Germany, France, Greece, and Austria, highlighting the treatment’s short- and long-term benefits in everyday medical practice.
“[Takhzyro] treatment can significantly reduce HAE attacks over a long duration,” the scientists wrote.
The observational study “Real-world Effectiveness of Lanadelumab in Hereditary Angioedema: Multi-country INTEGRATED Observational Study,” was published in The Journal of Allergy and Clinical Immunology: In Practice.
Takhzyro works to inhibit an enzyme needed to produce bradykinin
A rare genetic condition, HAE is marked by sudden swelling in the deeper layers of the skin and mucus membranes. These attacks, which can occur without warning, severely affect daily life and, if the airways are affected, can be life-threatening. Swelling attacks in HAE are caused by the overproduction of bradykinin, a signaling molecule.
Takhzyro is a lab-made antibody that’s designed to target and inhibit an enzyme that’s needed to produce bradykinin. By doing so, the therapy is expected to prevent or reduce the frequency of swelling attacks. Approved in the U.S. and European Union for patients ages 2 and older, Takhzyro can be administered to most patients once every two weeks. However, if swelling attacks are under control, dosing can be extended to once every four weeks.
Takhzyro’s efficacy in reducing HAE attack rates has been demonstrated in two Phase 3 clinical trials involving patients 12 and older and those ages 2 to 11, whose findings helped to support its approval. Here, researchers focused on assessing the therapy’s effectiveness in real-life clinical situations.
The study included 198 HAE patients, ages 12 and older (61% female; 91.9% with HAE type 1), who started treatment with Takhzyro in routine clinical practice between August 2018 and May 2021. Its main goal was to assess the effect of Takhzyro on the attack-free rate (AFR), defined as the proportion of patients who experienced no HAE attacks over a certain period of time.
Takhzyro’s use lowered the median attack rate by 98.8%, from a median of 2.2 attacks per patient each month before treatment to zero after the start of treatment, the researchers reported.
Monthly AFRs varied from 16.2% to 28.3% before treatment to 82.7% during the first month of treatment with Takhzyro, rising to more than 95% at various times between 26 to 43 months (about 2.2 to 3.6 years) of treatment.
“This real-world study demonstrates that [Takhzyro long-term treatment] is effective in improving AFR in HAE,” the researchers wrote, adding that its effectiveness was observed “starting from the first month of … therapy.”
Possibly of longer dosing times, lesser need for on-demand treatment
In addition to its effectiveness in preventing attacks, the study found that Takhzyro allowed for flexible dosing schedules. While all patients started by receiving an injection every two weeks, those who remained attack-free could have their dosing intervals extended to as much as eight weeks. In total, 144 of the 198 patients extended dosing after a mean 8.2 months of initial treatment: eight to once every 15 or 16 days, 38 to once every 17 or 18 days, 69 to once every three weeks, 25 to once monthly, one to once every six weeks, and three patients to once every two months. Some in this group, 35 patients, also returned to more frequent dosing.
Available data indicated the AFR per person-month remained high after a first dosing interval increase (95.1%), and the rate was similar to that before the start of an extended dosing period (91.5%).
According to the researchers, this finding “demonstrates that dosing frequency can be reduced while maintaining effectiveness in most of the patients.”
The study also explored Takhzyro’s impact on patients’ reliance on emergency treatments for HAE attacks. In the year before starting with Takhzyro, nearly all patients (99%) were using on-demand therapy. After beginning treatment with Takhzyro, the proportion of those with on-demand treatment dropped to 36.4% in the first year and to 52% across the entire follow-up.
“This study demonstrates the effectiveness of [Takhzyro long-term treatment] in improving AFR in HAE … patients [receiving treatment every two weeks] and dose interval increases,” the researchers wrote. They emphasized the importance of tailoring the therapy to a person’s needs, noting that “physicians are individualizing [Takhzyro] treatment by increasing the intervals of administrations in clinical practice.”
