Pompe Disease - Amicus Therapeutics

Student: Evan Cannon, 2019-2020

Sponsor: Amicus Therapeutics, Cranbury Township, NJ

Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering high-quality medicines for people living with rare and orphan diseases. Pompe disease is a fatal, inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glycosidase. It is a neuromuscular disorder that is characterized by severe muscle weakness that worsens over time. It ranges from a rapidly fatal infantile form with significant impact on heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle.

I am tasked with developing a global assessment of the commercial opportunity for a novel Pompe disease treatment being developed by Amicus. I will also be developing an unbranded mechanism of action awareness campaign to highlight the benefits of this treatment among physicians who treat Pompe patients.