Lanadelumab可有效降低遗传性血管性水肿患者的发病率

Introduction: Multiple studies have shown that lanadelumab can reduce the incidence of patients with type I or type II hereditary angioedema (HAE).

On February 28, 2022, Takeda published four abstracts, including real-world interim data from the observational Phase 4 EMPOWER study in North America of TAKHZYRO (lanadelumab) in patients with hereditary angioedema (HAE) type I or II; and HELP Open Label Post hoc analysis data from the Extension study, which evaluated the long-term safety and efficacy of TAKHZYRO in patients 12 years of age and older with HAE, were presented at the 78th Annual Meeting of the American Academy of Allergy, Asthma, and Immunology (AAAAI).

Key findings presented in the summary data include:

Scores on the Angioedema Quality of Life Questionnaire (AE-QOL), Angioedema Control Trial (AECT) and Treatment Satisfaction Questionnaire with Medications (TSQM-9), collected every 3 months, report on improvements in patients new to the medication and ongoing outcomes in patients on the older medication, as described in the Interim Data Presentation for EMPOWER IA2 Patient-Reported Outcomes.

As shown in interim data shared across the EMPOWER IA2 treatment modalities and subgroups, an average of 1 in 5 patients treated with TAKHZYRO was able to extend treatment from every 2 weeks to 4 weeks.

Real-world interim data show an 83% reduction in patient self-reported morbidity and no new safety signals on the efficacy and safety of EMPOWER IA2.

Post hoc analyzes of the HELP and HELP OLE trials showed that in the HELP 03 and HELP 04 studies of switching from androgen therapy to TAKHZYRO, the incidence (reduction) in patients who had previously received androgen therapy receiving TAKHZYRO was similar to the incidence in the broader population.

"We are pleased to see real-world data from EMPOWER showing improvements in angioedema control and treatment satisfaction," said Dr. Neil Inhaber, head of rare genetics and hematology, Global Medical Affairs, Takeda. "These interim results help better understand patients' overall experience with TAKHZYRO. Takeda has more than 10 years of experience and innovation in researching treatments for this devastating disease, and we remain steadfast in our commitment to providing better treatment options for patients with HAE."

In the observational Phase 4 EMPOWER study, real-world HAE incidence in patients with type I and type II HAE before and after treatment with TAKHZYRO is being evaluated. Full results from the EMPOWER study are expected to be published in 2024. HELP OLE is a completed Phase 3 study investigating the safety and efficacy of switching to TAKHZYRO in patients who had previously received androgens and other long-term preventive therapies.

In February 2018, the U.S. Food and Drug Administration (FDA) granted priority review status to lanadelumab for the prevention of angioedema attacks in patients 12 years of age and older. In August 2018, the FDA approved lanadelumab, becoming the first monoclonal antibody approved in the United States for the treatment of hereditary angioedema type I and type II (HAE) patients 12 years and older.

References:

https://www.takeda.com/newsroom/newsreleases/2022/interim-phase-4-data-support-takhzyro-lanadelumab-as-an-effective-treatment-to-reduce-attacks-in-hereditary-angioedema-patients/