Alglucosidase alfa

• Amicus Therapeutics will showcase 16 presentations at WORLDSymposium 2025, featuring comprehensive research on migalastat for Fabry disease and cipaglucosidase alfa plus miglustat for Pompe disease. • Key studies include long-term patient outcomes from the FollowME Fabry Pathfinders registry and real-world evidence of treatment effectiveness in both rare diseases. • Notable research highlights treatment satisfaction, quality of life improvements, and clinical outcomes in patients switching from existing therapies to Amicus' novel treatment approaches.

• Pompe disease, a rare genetic disorder, is seeing advancements with over 20 therapies in the pipeline. • Key players like Amicus Therapeutics and Spark Therapeutics are developing novel treatments, including gene therapies and enzyme replacements. • Clinical trials are underway, evaluating the safety and efficacy of drugs like Cipaglucosidase alfa and SPK-3006. • These emerging therapies target various mechanisms, such as alpha-glucosidase replacement and glycogen synthase kinase modulation.

• Analysis of 1,840 Health Technology Assessment decisions reveals that submissions including real-world evidence achieved 77% approval rate compared to 67% without RWE. • Case studies from Australia, Scotland, and France demonstrate how real-world evidence helped secure positive reimbursement decisions for drugs like Yervoy, Zaltrap, and Myozyme. • Despite proven benefits, real-world evidence was only utilized in 6% of HTA evaluations, suggesting significant untapped potential for both pharmaceutical companies and assessment agencies.