Fireside Chat, Interview, Conference Presentation
John F. Crowley, Chairman and CEO of Amicus Therapeutics
- The company anticipates a global launch for a new enzyme therapy, aiming to make patients switching from standard of care stronger with improvements in walking, talking, breathing, and quality of life.
- Regulatory submissions for approval are underway with FDA alignment, following a six-year development timeline from idea to clinic, with the company viewing the drug as potentially more effective and manufacturable than previous approaches.
- Material expectations include providing patient choices and alleviating human suffering by beating time, building on lessons from the COVID-19 response to accelerate research and break regulatory barriers.
- The outlook acknowledges risks including uncertainty during the pandemic that could cause anxiety about the company falling apart, alongside historical challenges where patients faced life-threatening conditions such as ventilator dependence and heart stopping.
- While the company may play a small role in the broader ecosystem, the plan involves a strong submission to ensure the medicine helps people breathe better, move arms better, and speak more clearly.