POMBILITI (cipaglucosidase alfa) / OPFOLDA (miglustat) - late-onset Pompe's disease

The Committee deems that the clinical benefit of POMBILITI (cipaglucosidase alfa) 105 mg powder for concentrate for solution for infusion in combination with OPFOLDA (miglustat) 65 mg hard capsules is low in the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency) in patients with prior enzyme replacement therapy.

Considering that:

• POMBILITI (cipaglucosidase alfa) in combination with OPFOLDA (miglustat) did not demonstrate any superiority compared to alglucosidase alfa (MYOZYME) combined with placebo on the primary endpoint of motor function, measured by the 6-minute walk distance test (6MWT) assessed after 52 weeks of treatment, in the total population included in the study, most of whom had received prior alglucosidase alfa therapy,
• according to the EPAR, the effect observed was considered to be clinically relevant because the minimum clinically important difference was around 21 metres in the cipaglucosidase alfa/miglustat group, in which most patients had received prior alglucosidase alfa therapy,
• the safety profile of the POMBILITI (cipaglucosidase alfa) and OPFOLDA (miglustat) combination appears to be favourable,
• the medical need is partially met in patients who have not responded to enzyme replacement therapy in this rare and debilitating disease,

the Committee deems that POMBILITI (cipaglucosidase alfa) 105 mg powder for concentrate for solution for infusion and OPFOLDA (miglustat) 65 mg hard capsules provide no clinical added value (CAV V) compared to MYOZYME (alglucosidase alfa).