Clinical outcomes of two new generation enzyme therapies in Pompe disease

• The results of two 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. clinical trials evaluating new-generation double-blind enzymeProtéine capable d’activer une réaction biochimique précise. therapies in late-onset Pompe disease against Myozyme were published in December 2021. 

• Both treatments were well tolerated and they preserved or even improved patients’ walking and breathing capacity after one year of treatment. 

• Avalglucosidase alfa (Nexviadyme), evaluated in the COMET trial in 100 enzyme-naïve participants, showed non-inferiority to Myozyme over one year. The authors point out that several clinical parameters support avalglucosidase alfa. The extension of the trial will allow the evolution of the therapeutic response to be followed over time. In France, avalglucosidase alfa is available under temporary authorization for use. 
• Cipaglucosidase alfa, combined with miglustat (AT-GAA) was evaluated in the PROPEL trial, both in people already treated with Myozyme and in people naïve to any enzyme therapy. As with avalglucosidase alfa, it is not possible to conclude on the superiority of the treatment compared to Myozyme, except for patients previously treated with Myozyme.

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Jordi Diaz-Manera, Priya S Kishnani, Hani Kushlaf et al. Lancet Neurol. 2021 (Déc)

 

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial. Benedikt Schoser, Mark Roberts, Barry J Byrne et al. Lancet Neurol. 2021 (Déc)

 

New therapies for Pompe disease: are we closer to a cure? Rosa Puertollano, Nina Raben. Lancet Neurol . 2021 (Déc)

 

Voir aussi « Maladie de Pompe : l’arsenal thérapeutique s’agrandit »