TAKHZYRO (lanadelumab) - Hereditary angioedema

Opinions on drugs - Posted on Mar 05 2024 - Updated on Jun 26 2024

Reason for request

First listing

Key points

Favourable opinion for reimbursement in “routine prevention of recurrent attacks of hereditary angi oedema (HAE) in patients aged from 2 years to less than 12 years.”

Clinical Benefit

Substantial

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.

Clinical Added Value

moderate

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.

minor

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 (with the majority of patients (81%) aged from 6 years to under 12 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, compared with the alternative, CINRYZE (C1 esterase inhibitor), which is administered by the IV route every 3 to 4 days,
but the absence of comparison versus the clinically relevant comparator CINRYZE (C1 esterase inhibitor), despite the fact that such a comparison would have been possible,

the Committee deems that TAKHZYRO (lanadelumab) 150 mg provides a minor clinical added value (CAV IV) in the prevention of recurrent attacks of hereditary angioedema in patients aged 6 years to 11 years.