On January 20, 2021, Mallinckrodt Pharmaceuticals called Phil Marella of DART and other NPC community leaders and advised us that they were not going to go forward with an FDA new drug application for Adrabetadex/Cyclodextrin. Of course, this is devastating and frightening news for the Marella family and the more than 80 children in the US benefiting from the drug. Andrew Marella starting in the phase 1/2 clinical trial of the drug over 7 years ago and its benefit has been nothing short of miraculous.
Working with the Ara Parseghian Fund, the National Niemann-Pick Disease Foundation and other NPC family charities, DART will work tirelessly to keep this amazing therapy available to patients and hopefully find a new path to FDA approval.
