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2024 AAAAI HAE Conference Coverage

CE / CME

Key Updates on Hereditary Angioedema: CCO Independent Conference Coverage of the 2024 AAAAI Annual Meeting

Physician Assistants/Physician Associates: 0.50 AAPA Category 1 CME credit

Nurses: 0.50 Nursing contact hour

Physicians: maximum of 0.50 AMA PRA Category 1 Credit™

Released: March 22, 2024

Expiration: March 21, 2025

CME/CE Information

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EMPOWER: Long-term Efficacy and Safety of Lanadelumab in HAE

Next, I discuss SC lanadelumab, an mAb that targets plasma kallikrein and is approved by the FDA for long-term prophylaxis to prevent HAE attacks in adult and pediatric patients aged 2 years or older. Although this medication has been used for the past several years, we continue to see important data on lanadelumab to assess its efficacy and safety with long-term use.9

Two phase IV trials were presented at AAAAI 2024, so these were postmarketing observational studies. First is the EMPOWER trial that evaluated the long-term efficacy and safety of lanadelumab in patients aged 12 years or older with type I or II HAE who were new or prevalent users. New users were defined as having received <4 doses of lanadelumab before enrollment; prevalent users were defined as having received ≥4 doses of lanadelumab with the last dose <70 days before enrollment. Enrolled patients were from the United States or Canada and received treatment for ≥36 months.

In total, 113 patients were enrolled. The early period was defined as the first 69 days of treatment, followed by the steady-state period (after 70 days of therapy). Efficacy was assessed as change in HAE attack rate per person-month, evaluated before and after treatment initiation. Safety endpoints included nonserious and serious TEAEs.10

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EMPOWER: Baseline Characteristics

Most patients (82%) were deemed prevalent users, and 20 patients were evaluated, in particular for efficacy, as new users. Overall, the majority (>65%) were women, White, and diagnosed with type I HAE. Approximately 19% of patients were diagnosed with type II HAE.10

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EMPOWER: Lanadelumab Efficacy

The efficacy evaluation focused on new users (n = 20). Prior to treatment, patients experienced a mean 1.57 HAE attacks per month. Then, in the early-state period, lanadelumab resulted in a 60% reduction. Furthermore, an 80% reduction in HAE attacks was observed in the steady-state period and cumulative timeline.10

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EMPOWER: Lanadelumab Safety

Regarding safety, very few AEs (4.7%) were related to lanadelumab use. These events included fatigue, worsening fatigue, tachycardia, worsening tachycardia, and bilateral papular pruritic rash—all of which were reported by 2 patients. Of interest, no major issues with injection-site reactions were observed. No TEAEs were serious in severity or led to treatment discontinuation.10

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Long-term Lanadelumab Efficacy and Safety for HAE in Adolescents: From ENABLE and EMPOWER

Additional lanadelumab data presented at AAAAI 2024 focused on long-term efficacy and safety in adolescents (aged 12 to <18 years) from a pooled analysis of the noninterventional, prospective phase IV ENABLE and EMPOWER trials. This is an important cohort of patients because HAE can worsen or become more prominent, specifically with symptoms, during the teenage years.11 Furthermore, there are relatively few data from clinical trials dedicated to this patient population.

EMPOWER was held in North America, and ENABLE was largely conducted across several countries including Austria, Germany, Kuwait, Israel, Italy, Spain, and Switzerland. Therefore, this pooled analysis provides real-world data on long-term lanadelumab in adolescents with HAE.

Again, the strategy of stratifying new vs prevalent users was applied. New users were defined as having received <4 doses before enrollment; prevalent users were defined as having received ≥4 doses with the last dose <70 days before enrollment. In total, 20 patients were included in this analysis, with 13 new and 7 prevalent users.

Efficacy was evaluated as change in HAE attacks rate per person-month, and safety outcomes included TEAEs or serious AEs.12

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Lanadelumab Efficacy in Adolescents: EMPOWER and ENABLE

In new users, lanadelumab resulted in a 63% reduction in HAE attacks. Of the available data, most attacks affected either the patient’s abdomen or periphery. These were largely moderate attacks (71%), with some mild (8%) or severe (22%) events recorded. More than 90% received some acute treatment for their attack.

In prevalent users, the mean monthly attack rate was 0.10. Most attacks affected patients’ abdomen and had an unknown severity. Some (23%) were moderate, and few (14%) were mild or severe. More than 80% received some acute treatment for their attack.12

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Lanadelumab Safety in Adolescents: EMPOWER and ENABLE

In terms of safety, there were 41 TEAEs across both new and prevalent user cohorts. None was deemed to be related to lanadelumab use. Furthermore, no TEAE resulted in discontinuation or death.

There were only 3 serious TEAEs, experienced by 2 patients in the new user cohort. Most TEAEs across both groups were mild or moderate. We are not seeing any differences in the adolescent population compared with what has been described in other data sets.12

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Long-term Lanadelumab for HAE: Summary

This newly presented data on lanadelumab showed both safety and efficacy that was consistent with what is seen in prior studies. Furthermore, lanadelumab resulted in significant reductions in HAE attack rates, including for these long-term observation periods (≤36 months after initiation).

Data on adolescents show very comparable efficacy and safety that has been seen in the other studies of the adult population. In addition, there were no TEAE related to lanadelumab that led to study discontinuation. While focused on the adolescent population, this remains a small study. Unfortunately, it is difficult to get large data sets on adolescents, so I think these trends are quite helpful for us clinically.10,12

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