U.S. Expanded Access Program to Continue,
September 16, 2021

To The NPC1 Community,

We are thrilled today to announce that we’ve reached agreement with the FDA to officially continue the US Expanded Access Program (EAP) for intrathecal administration of adrabetadex* beyond October 20th, 2021.

We recognize the anxiety, concern, and frustration caused by the uncertainty related to this program prior to our acquisition of adrabetadex, and we hope that this announcement will reduce at least some of the tension that patients, caregivers, families, and physicians have been experiencing for the past nine months.

We still have much work to do and will be in touch again soon with further updates.


Scott Riccio
EVP, Patient & Community Engagement
Mandos, LLC