We concentrate our research efforts where medical needs and public health issues are, bringing solutions to patients and contributing to a healthier society worldwide.
For an overview of the current Sanofi Rare Diseases Pipeline, view our full list of investigational agents Click here.
*Avalglucosidase alfa has received marketing authorization in several countries for infantile-onset Pompe disease (IOPD) and/or
late-onset Pompe disease (LOPD). In the United States, it was approved in August 2021 for patients with LOPD ≥1 year of age and, in the
European Union (EU), it was approved in June 2022 for the treatment of patients with Pompe disease. The other agents mentioned here
are investigational and have not been approved by the US Food and Drug Administration (FDA), European Medicines Agency (EMA) or any
other regulatory agency worldwide for their uses under investigation.
AAV, adeno-associated virus; FGFR3, fibroblast growth factor receptor 3; IOPD, infantile-onset Pompe disease; mAb, monoclonal antibody; PAH, phenylalanine hydroxylase; TGFb, transforming growth factor-.
Clinicaltrials.gov: 1. NCT04910776 2. NCT05222906 3. NCT05206773, NCT05280548 4. NCT05856331; 5. NCT06500702.
The Role of GLYCOSPHINGOLIPIDS and SPHINGOLIPIDS in Rare Lysosomal Storage Diseases and other Rare Diseases
____________________________________________________________________________________________________________________________________
Clinical Studies in Patients with Infantile-Onset Pompe disease (IOPD) Clinical Trial Brochure
