Sanofi and its specialty care global business unit Sanofi Genzyme presented data from NEO1, its Phase 1/2 clinical study evaluating the investigational novel enzyme replacement therapy neoGAA in 24 patients with late-onset Pompe disease.
The safety and efficacy data from this study, which were presented at WORLDsymposium 2016 in San Diego, CA, support further development of the therapy. Sanofi Genzyme plans to begin enrolling patients in a pivotal Phase 3 trial for neoGAA in Q2 2016.
Pompe disease is a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA) affecting an estimated 50,000 people worldwide. Patients often lose their ability to walk and require wheelchairs to assist with mobility. They also often experience difficulty breathing and may require mechanical ventilation to breathe.
