Amicus Therapeutics
Amicus Therapeutics is a biotechnology company dedicated to developing advanced therapies for a range of devastating rare diseases. Driven by innovation, integrity, and a patient-focused mindset, the company aims to transform the lives of those living with rare and orphan diseases through scientific excellence and strategic partnerships.
Industries
Nr. of Employees
large (251-1000)
Amicus Therapeutics
Patents
Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
US-12275703-B2
View Details
Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
US-12275703-B2
View DetailsProducts
Migalastat (oral small-molecule therapy for Fabry disease)
An orally administered small-molecule therapeutic indicated for the treatment of Fabry disease in patients with eligible genetic variants.
Cipaglucosidase alfa combined with miglustat (enzyme replacement therapy for Pompe disease)
An enzyme replacement therapy co-administered with an uptake-enhancing small molecule intended for the treatment of Pompe disease.
Patient support and access program
A patient assistance and support service to help patients access therapies and related resources.
Migalastat (oral small-molecule therapy for Fabry disease)
An orally administered small-molecule therapeutic indicated for the treatment of Fabry disease in patients with eligible genetic variants.
Cipaglucosidase alfa combined with miglustat (enzyme replacement therapy for Pompe disease)
An enzyme replacement therapy co-administered with an uptake-enhancing small molecule intended for the treatment of Pompe disease.
Patient support and access program
A patient assistance and support service to help patients access therapies and related resources.
Services
Provision of expanded access pathways to investigational or approved therapies for eligible patients.
Management of patient advisory boards, resources and advocacy initiatives to incorporate patient perspectives into development and access.
Global medical strategy, scientific communication and support for clinical and commercial activities.
Management of manufacturing strategy, CMC oversight, external manufacturing relationships and supply chain operations.
Strategic portfolio planning, alliance management and program analytics to prioritize development and commercial activities.
Provision of expanded access pathways to investigational or approved therapies for eligible patients.
Management of patient advisory boards, resources and advocacy initiatives to incorporate patient perspectives into development and access.
Global medical strategy, scientific communication and support for clinical and commercial activities.
Management of manufacturing strategy, CMC oversight, external manufacturing relationships and supply chain operations.
Strategic portfolio planning, alliance management and program analytics to prioritize development and commercial activities.
Expertise Areas
- Rare disease therapeutics development
- Clinical trial management
- Gene therapy development
- CMC and biologics manufacturing
Key Technologies
- Gene therapy
- Enzyme replacement therapy
- Small-molecule rare-disease therapeutics
- Chemistry, Manufacturing and Controls (CMC) processes
News & Updates
John F. Crowley, Chairman Emeritus, delivered a commencement address at Notre Dame, emphasizing hope, science, and dedication to rare disease communities.
John F. Crowley, Chairman Emeritus, delivered a commencement address at Notre Dame, emphasizing hope, science, and dedication to rare disease communities.