COMET clinical trial in Pompe disease: first results show a beneficial effect of neoGAA

The COMET clinical trial compared the efficacy and safety of neoGAA, a new generation enzyme therapy, with Myozyme, a standard treatment in Pompe disease, both developed by Sanofi-Genzyme. This double-blind, phase 3 trial involved 100 patients with late-onset disease in 56 different centers in 20 countries around the world.
Preliminary analysis of the data after one year (49 weeks) shows in patients treated with neoGAA:

  • a 2.4 point improvement in the percentage of theoretical vital capacity;
  • a 30 meter improvement in the distance covered in the 6 minute walk test.

Sanofi-Genzyme intends to compile a marketing authorization file with the various competent health authorities by the end of the year.


Access Sanofi-Genzyme press release 16 June 2020 “Sanofi’s investigational enzyme replacement therapy shows clinically meaningful improvement in critical manifestations of late-onset Pompe disease