Welcome to the International Pompe Association (IPA)
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 becompleted 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.
CRANBURY, N.J., May 15, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC). Consistent with previous results presented at the 2017 WORLDSymposium™, patients treated with ATB200/AT2221 continue to show improvements in biomarkers of muscle damage and disease substrate.
"We are very pleased to see improvements in six minute walk distance and other measures of motor function in both naïve and ERT-switch patients, as well as stability or improvements in forced vital capacity. The consistency and magnitude of improvements exceeded our expectations and follow the initial improvements seen on key biomarkers of muscle damage and disease substrate," said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "These preliminary functional results are very encouraging and suggest a clinically meaningful improvement for patients. We look forward to additional data from all patients in the third quarter as we continue in our mission to develop an improved treatment option for people living with Pompe disease."...
Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.
The IPA Board was extremely saddened to learn of the unexpected passing of Henri Termeer, former CEO of Genzyme. Every tribute to Henri has mentioned the patient-centered focus he instilled and fostered within Genzyme. From personal experience, the IPA fully agrees with this characterization. The Pompe community has been extremely fortunate to have had Genzyme and Henri Termeer as partners in developing a treatment for Pompe. In the Pompe Community we have a true partnership between the three stakeholders: the Medical/Scientific Community, Industry, and the Patient Community. This partnership is only possible when all parties respect and value each other. The IPA believes that Henri Termeer was instrumental in setting the tone for Industry partners to engage with, and learn from their counter-parts in the Patient Community. He will be greatly missed.
- Henri Termeer, key biotech leader who built Genzyme into an industry giant, dead at 71 (The Boston Globe)
- Bio Leaders Mourn Passing of Genzyme Exec, Orphan Drug Innovator Termeer (xconomy.com)
- Henri Termeer Obituary: Link 1, Link 1