TAKHZYRO (lanadelumab) - Hereditary angioedema

The Committee deems that the clinical benefit of TAKHZYRO (lanadelumab) 150 mg solution for injection in pre-filled syringe is substantial in the preven tion of recurrent attacks of hereditary angioedema (HAE) in patients aged from 2 years to less than 12 years. 

Considering: 

• the suggested efficacy of TAKHZYRO (lanadelumab) in a non-comparative phase 3 study in  21 patients with a median age of 8.7 years (min 3.5 - max 10.9 years) over a significant period of 52 weeks, with a marked reduction in the number of attacks per month, decreasing from 1.8 to 0,08, i.e. a 94.8% reduction, and the number of severe attacks, decreasing from 1.27 to 0.08, i.e. a reduction of 96.8%, 
•  a safety profile marked primarily by pain at injection site, skin abrasions and headaches, and which appears to be similar to that observed in older patients, 
•  the convenience of TAKHZYRO (lanadelumab), with subcutaneous administration every 2 to 4 weeks,
• the unmet medical need in the absence of a therapeutic alternative in these patients and the uncertain efficacy of tranexamic acid used off-label, 

the Committee deems that TAKHZYRO (lanadelumab) 150 mg provides a moderate clinical added value (CAV III) in the prevention of recurrent attacks of hereditary angioedema in patients aged 2 years to less than 6 years.