Orphazyme has an Early-Access Program (EAP) in the United States, France and Germany for people living with Niemann-Pick disease type C (NPC). EAPs are sometimes referred to as Expanded 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 these markets.
Requesting Early Access to Arimoclomol for Patients with NPC
Orphazyme has partnered with the Clinigen Group to coordinate arimoclomol Early Access.
United States: US Healthcare Professionals can obtain details about the arimoclomol EAP or make a request for early access for their patient by calling Clinigen customer service team at +1 877-768-4303 or emailing
To see the list of US active sites, please visit the trial information on clinicaltrials.gov – https://clinicaltrials.gov/ct2/show/NCT04316637
European Union: EU Healthcare Professionals can obtain details about existing Early Access to arimoclomol from publicly available information provided by the competent regulatory authority. To obtain additional information or
make a request for Early Access for their patient, healthcare professionals should contact Clinigen customer service team at:
Please note that the initial request should not contain personally identifiable information about the patient (e.g. name, contact details, personal identification number). Receipt of the request will be acknowledged within five business days.
Patients seeking medical information should either contact their physician or email@example.com.