Posted by Michael Wonder on 18 Feb 2026
NICE recommends first ever disease modifying treatment for ARG1 deficiency
17 February 2026 - NICE has recommended the first disease modifying treatment for arginase 1 deficiency (ARG1-D), an ultra-rare, inherited and progressive metabolic disorder, for routine use in the NHS in England.
In our final draft guidance, we’ve recommended pegzilarginase (Loargys, Immedica) for treating ARG1-D in adults, adolescents and children aged 2 years and older.
