Favourable opinion for reimbursement in the “treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older”.
Clinical Benefit
Substantial
The Committee deems that the clinical benefit of LOARGYS (pegzilarginase) is substantial in the treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older.
Clinical Added Value
minor
Considering:
demonstration of the superiority of LOARGYS (pegzilarginase) for the primary endpoint, which was a major biological endpoint for treatment follow-up, i.e. change in plasma arginine at 24 weeks compared to placebo, both combined with individualised disease management (dietary protein restriction ± nitrogen scavengers) in a phase 3, randomised, double-blind trial conducted in 32 patients with arginase 1 deficiency, aged from 2 to 29 years (mean age 10.7 years),
the safety profile of LOARGYS (pegzilarginase), which seems to be favourable with limited follow-up,
the absence of evidence of a benefit of LOARGYS (pegzilarginase) on neurocognitive or motor impairment,
exploratory results concerning simplification of background therapy, which is particularly restrictive, in parallel with plasma arginine levels,
exploratory results concerning quality of life in this disease with a significant impact on quality of life,
the Committee deems that LOARGYS (pegzilarginase) 5 mg/ml solution for injection/infusion provides a minor clinical added value (CAV IV) in the current care pathway.
Listen
