LOARGYS (pegzilarginase) - Arginase 1 deficiency (ARG1-D)

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.

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.