FDA approves lanadelumab for hereditary angioedema in patients aged 2 years and older

The US Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for lanadelumab (TAKHZYRO; Takeda) for expanded use in pediatric patients aged 2 to under 12 years at risk of hereditary angioedema (HAE) attacks.

HAE attacks can lead to severe swelling in the face, throat, hands, feet, genitals, and abdomen. Patients aged as young as 3 years may experience fatal upper airway angioedema. About 1 in 50,000 people worldwide experience HAE, which is often under-diagnosed and under-treated.

A survey from 2017 showed a mean 8.4 years between symptom onset and HAE diagnosis. The study cohort with HAE showed anxiety in 50% of patients, difficulty with social activity in 34%, and symptoms negatively affecting career advancement in 58%.

Prior treatment options against HAE attacks had been approved in children aged 6 to under 12 years, but options were not available for children aged 2 to under 6 years. Lanadelumab is the first drug for preventing HAE attacks in this age group.

The recommended dose of lanadelumab is 150 mg/1 mL, delivered through a prefilled syringe once every 4 weeks for patients aged 2 to under 6 years. Patients aged 6 to under 12 years are recommended to take 1 dose every 2 weeks.

The approval came following the SPRING study, a phase 3 trial evaluating the safety and efficacy of lanadelumab in pediatric patients aged 2 to 12 years. Common adverse effects from lanadelumab in this population included site pain, injection site erythema, injection site swelling, administration site pain, and injection site reaction.

HAE attacks saw a 94.8% reduction in pediatric patients taking lanadelumab when compared to baseline. This led from 1.84 attacks per month to 0.08 attacks over 52 weeks of treatment. About 76% of patients experienced no attacks, with 99.5% attack-free days experienced on average.

Lanadelumab decreases plasma kallikrein activity to reduce HAE attacks. While the drug can be self-administered in patients aged 12 years and older, patients aged 2 to under 12 years should have dosages administered by a healthcare provider or trained caregiver. 

Potential severe adverse events include wheezing, difficulty breathing, chest tightness, fast heartbeat, faintness, rash, and hives. Patients should receive emergency help or contact a healthcare provider if they are experiencing any of these symptoms.

Reference

US FDA approves Takeda’s TAKHZYRO (lanadelumab-flyo) to prevent hereditary angioedema (HAE) attacks in children 2 years of age and older. Business Wire. February 3, 2023. Accessed February 6, 2023. https://www.businesswire.com/news/home/20230203005431/en/U.S.-FDA-Approves-Takeda%E2%80%99s-TAKHZYRO%C2%AE-lanadelumab-flyo-to-Prevent-Hereditary-Angioedema-HAE-Attacks-in-Children-2-Years-of-Age-and-Older