Clinical stability in real-world settings following a switch to new enzyme therapies in Pompe disease

Although phase IIIAu cours d’un essai clinique de phase III, un médicament, pour lequel on a déterminé lors d’essais antérieurs l’innocuité et le dosage optimum (essais de phase I et II), est administré à un grand groupe de malades, sur une longue durée, dans le but d’évaluer son efficacité thérapeutique en la comparant à celle d’un traitement de référence ou un placebo. Il permet aussi de mettre en évidence les interactions indésirables et les effets secondaires du traitement à moyen terme. Au terme de cet essai, le médicament peut obtenir une autorisation de mise sur le marché. trials (the COMET and PROPEL trials) have demonstrated the efficacy of next-generation enzymeProtéine capable d’activer une réaction biochimique précise. therapies – avalglucosidase alfa (Nexviadyme®) and cipaglucosidase alfa (Pombiliti®) – in Pompe disease, real-world data remain limited.

A prospective study conducted at five centres in Germany, where both treatments are available, analysed data from 39 adults with late-onset Pompe disease who switched from alglucosidase alfa (Myozyme®) to one of the two new therapies.

• Respiratory function remained stable and walking tests showed no significant decline up to two years after switching enzyme replacement therapy, suggesting that switching may be a useful strategy, particularly in cases of efficacy issues.
• Serious or injection-related adverse events were rare.
• However, interpretation of the results was severely limited by incomplete follow-up data, particularly beyond one year.
• Adherence to the European Pompe Consortium (EPOC) assessment guidelines was variable, which introduced heterogeneity and complicated comparisons between centres.

Systematic long-term data collection via patient registries and standardised assessments will be essential for better identifying the patients most likely to benefit from a change in treatment.

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease. Mendelsohn D, Rosenbohm A, Güttsches A et al. J Neurol. 2026 Feb.

 

Voir aussi « Maladie de pompe à début tardif : un changement d’enzymothérapie est possible »