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.
**Imiglucerase is indicated for treatment of adults and pediatric patients 2 years of age and older with type 1 Gaucher disease and is being evaluated for type 3 Gaucher 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; BTK, bruton's tyrosine kinase; CD, cluster of differentiation; IOPD, infantile-onset Pompe disease; mAb, monoclonal antibody; TNF-α, tumor necrosis factor-alpha.
Clinicaltrials.gov: 1. NCT04910776; 2. NCT00358943; 3. NCT05222906; 4. NCT05206773, NCT05280548; 5. NCT06844214; 6. NCT05856331; 7. NCT06500702.
Clinical Studies in Patients with Infantile-Onset Pompe disease (IOPD) Clinical Trial Brochure
Result Trial Brochure
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RESULT Study Design Video
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RESULT Example of Potential Eligible Patients