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NeoGAA, a new generation of enzyme replacement therapy developed by Sanofi Genzyme will be tested in 96 patients with type 2 glycogenosis (Pompe disease). The recruitment of this trial is ongoing.

This international trial takes place in more than 20 different countries.

The aim is to compare the safety of use and efficacy of neoGAA with the only treatment currently available in Pompe disease. For this, 96 people who have not been previously treated for Pompe Disease, will be treated for 1 year, either by neoGAA or by Myozyme®. After this first year, all patients will receive NeoGAA for about 2 years. This clinical trial began in October 2016 and is expected to be completed by 2020.

A new generation recombinant enzyme

NeoGAAA and Myozyme® are recombinant forms (created by genetic engineering) of human acid alpha-glucosidase (GAA), the defective enzyme in Pompe disease. Myozyme® has been used since 2006 in the treatment of Pompe disease.

NeoGAA is a drug candidate developed from Myozyme® to increase its effectiveness. It has already been tested in humans in a phase 1/2 trial (NEO1), conducted in 24 people with Pompe disease for 6 months. Based on first results from this trial, the European Medicines Agency has attributed this molecule an orphan drug status. This designation applies to drug candidates (who have not yet proved their effectiveness) in rare diseases, in order to facilitate the different stages of their development.

To participate in the trial

The trial takes place in more than 75 different centers around the world. Individuals wishing to apply to participate in this trial must meet a number of criteria and obligations related to the trial protocol (to ensure valid and reliable conclusions). Among the main criteria, one must be suffering from Pompe disease, be over 3 years old and have never received a Myozyme® infusion.

If you have any questions or would like to participate in this trial as a patient, please contacty our treating physician.

Information on all clinical trials can be found at (search “Pompe”).

Link to this study on Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET).


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