Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHSScotland for the treatment of Pompe disease
The SMC has accepted cipaglucosidase alfa as a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).
Source:
Scottish Medicines Consortium
SPS commentary:
NICE has recommended cipaglucosidase alfa plus miglustat as an option for treating late-onset Pompe disease in adults (TA912).