Developing a commercial launch strategy for a novel therapeutic treatment for Niemann Pick Disease Type C (NPC)

Student: Fiona McDonnell, 2019-2020

Sponsor: Kevin Vaughan, Notre Dame, IN

Niemann Pick Disease Type C (NPC) is an ultra-rare, genetic, lysosomal storage disorder that results in accumulation of cholesterol and lipids within cells. This accumulation is toxic to cells and leads to an increased levels of cell death in patients. The resulting symptoms start to manifest in early childhood and include liver disease, poor muscle tone, speech and swallowing issues, and decline in intellectual function. Neurodegeneration also occurs and patients usually die from the disease prior to reaching adolescence. 

To-date, there is no FDA approved therapy for NPC. I will be working with a Notre Dame research group to understand the commercial opportunity for a new therapeutic drug for this fatal disease. My project will focus on deep diving into the technology, looking at the potential markets for this novel therapeutic, completing a competitive landscape assessment, understanding the international regulatory barriers associated with bringing a new therapeutic product to market and mapping out a commercial launch strategy for this treatment.