Orphazyme is focused on the treatment of rare neurodegenerative diseases by harnessing the cell protective properties of the heat shock response, a natural defense mechanism system present in all our cells.
We are committed to making every effort to support patients in need of treatment options.
Our clinical studies evaluate the potential of our investigational treatments in a well-defined patient population with eligibility criteria that allow for appropriate assessment of safety and clinical effect. If you are interested in learning more about our ongoing clinical studies, please contact us at medicalaffairs@orphazyme.com.
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.
Orphazyme has partnered with the Clinigen Group to coordinate arimoclomol Early Access.
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 medicalaffairs@orphazyme.com.