Expanded Access Program

Orphazyme has an Expanded Access Program (EAP) in the United States for people living with Niemann-Pick disease type C (NPC). EAPs are sometimes referred to as Early Access Programs, compassionate use or similar locally defined pre-approval access programs. We expect the EAP to remain open until the investigational treatment arimoclomol becomes commercially available in the US.

To see the list of active sites, please visit the trial information on clinicaltrials.gov – https://clinicaltrials.gov/ct2/show/NCT04316637.

We have partnered with Clinigen Group to administer the NPC EAP. Healthcare providers can obtain details about the NPC EAP by calling the Clinigen customer service team at + 1 877-768-4303 or emailing the Clinigen directly at usmapoperations@clinigengroup.com.



Orphazyme is reimagining scientific congresses to showcase progress across its therapeutic focus areas and platforms. This section brings together the newest resources around medical congresses and events.

WORLDSymposium (February 8-12, 2021)
MDA Virtual Clinical & Scientific Congress (March 15-18, 2021)
ACMG Annual Clinical Genetics Meeting (April 13-15, 2021)