The CHMP said late last week it was recommending Pombiliti (cipaglucosidase alfa or ATB200) as an infused therapy for late-onset Pompe disease in adults given in combination with the enzyme stabiliser miglustat in a regimen known as AT-GAA.
It’s the first of two regulatory decisions needed by Amicus to bring the new therapy to the EU market, as the CHMP is due to deliver a verdict on miglustat in the first quarter of 2023, according to company estimates.
