This segment focuses on the development, manufacturing, and commercialization of treatments for Fabry disease, a rare genetic disorder. The primary product in this segment is Galafold (migalastat), an oral precision medicine. Research and development activities include ongoing clinical trials to evaluate the efficacy and safety of Galafold in various patient populations and exploring new formulations or delivery methods. The segment utilizes advanced technologies such as in vitro assays to identify amenable GLA gene variants. Patient impact is significant, as Galafold provides a treatment option for adults with Fabry disease, potentially slowing disease progression and improving quality of life. Market positioning is strong due to Galafold's unique mechanism of action and oral administration. Future opportunities include expanding the label to include additional patient populations and geographic regions. Regulatory aspects involve obtaining and maintaining marketing authorizations globally. Partnerships and collaborations are crucial for manufacturing and distribution.

This segment is dedicated to developing novel therapies for Pompe disease, a rare genetic disorder characterized by the buildup of glycogen in the body's cells. Amicus is developing AT-GAA, a novel treatment paradigm for Pompe disease. Research and development efforts include clinical trials to assess the safety and efficacy of AT-GAA and other enzyme replacement therapies. The segment leverages advanced technologies in protein engineering and gene therapy. The goal is to provide effective treatments that improve the lives of patients with Pompe disease by addressing the underlying cause of the disease. Market positioning is focused on providing innovative treatment options for this underserved patient population. Future opportunities include expanding the clinical development program and exploring combination therapies. Regulatory aspects involve obtaining approvals from regulatory agencies. Strategic partnerships are essential for manufacturing and commercialization.

This segment focuses on the development of gene therapies for rare neurological disorders, specifically CLN3 disease (Batten disease) and CDKL5 deficiency disorder. Research and development activities include Phase 1/2 clinical studies to evaluate the safety and efficacy of AAV serotype AT-GTX-502 gene therapy for CLN3 and exploring gene therapy approaches for CDKL5. The segment utilizes advanced gene therapy technologies, including adeno-associated viral (AAV) vectors. The aim is to provide potentially curative treatments for these devastating diseases, improving neurological function and quality of life for patients. Market positioning is focused on addressing significant unmet medical needs in rare neurological disorders. Future opportunities include expanding clinical trials and exploring the potential of gene therapy for other neurological conditions. Regulatory aspects involve navigating the complex regulatory pathways for gene therapy products. Partnerships and collaborations are crucial for manufacturing and clinical development.