Together, we will beat NPC!

The NPC community is experiencing unprecedented investment and progress in developing treatments for the disease. Today, a number of companies have NPC programs underway, two of which are in later stages of regulatory review with the FDA. Additionally, there are a number of preclinical programs being funded and supported by NPC organizations, government grants, or investors. There are also a number of collaborative research programs established to tackle key aspects of the disease that are being managed by various organizations and research groups.

The Foundations, families, and patient advocacy organizations play an increasingly critical role in supporting successful regulatory and access strategies, and helping researchers and companies navigate the NPC space. The NPC Community demonstrated its ability to coordinate and convene strategic efforts in the 2019 Patient-Focused Drug Development meeting, as well as the related patient preference surveys and engagement with the FDA. The opportunity exists to build on this collaborative cornerstone with a focused set of actions to reduce the time to market approval for NPC therapies and support patient access, while at the same time coordinate with the FDA and keep families at the front and center of each program.

Building off these successes, we are launching the NPC Therapy Acceleration (NTA). This collaborative initiative will be for the entire NPC community and will be guided by the patient perspective. The NTA will work with and gather relevant information from NPC companies and preclinical research groups and help them align with the needs of the patient. It will also connect companies and researchers to key NPC scientists, labs, tools, provide mentorship and even connect to potential investors. Additionally, the collaboration will be a place to share relative data from the many initiatives ongoing in the NPC community. The NTA will support a forum to help families with access to health care and will largely be managed by patient advocacy organizations. Finally, all aspects of the NTA will be coordinated with the FDA and relative government regulatory agencies.

Learn more about the Accelerator ›

Meet the team ›