Cipaglucosidase alfa, or Pombiliti®, is a recombinant enzyme being tested in Pompe disease in combination with miglustat (Opfolda®). The combination of the two drug candidates was evaluated in the phase III PROPEL trial involving 118 patients with the late form of the disease. Following an initial one-year phase against treatment with Myozyme® in combination with placebo, a one-year open-label extension phase was conducted.
- The results at two years show an improvement in walking ability and stabilisation of respiratory capacity thanks to the treatment.
- Safety was similar to that of Myozyme®.
Despite marketing authorisation in Europe, the treatment is not yet available in France.
