Orphazyme A/S (ORPHA.CO), a biopharmaceutical company pioneering Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces the availability of an Early Access Program (EAP) in the United States (US) for its investigational drug arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).The EAP provides a pathway for patients with serious, life-threatening diseases or conditions who lack therapeutic alternatives to gain access to investigational drugs before they are approved.Kim Stratton, Chief Executive Officer, commented, Orphazyme is deeply committed to providing access to new therapies for people living with rare diseases such as NPC. There are currently no approved products to treat NPC in the US and we are pleased to make arimoclomol, a potential first-in-class innovative oral therapy, available pre-commercially to US patients through this program. We planto file for approval of arimoclomol in the US in H1 2020.
Orphazyme A/S (ORPHA.CO), a biopharmaceutical company pioneering Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases,
today announces the availability of an Early Access Program (EAP) in the
United States (US) for its investigational drug arimoclomol for the
treatment of Niemann-Pick disease Type C (NPC).The EAP provides a pathway
for patients with serious, life-threatening diseases or conditions who lack therapeutic alternatives to gain access to investigational drugs before they are approved.Kim Stratton, Chief Executive Officer, commented, Orphazyme is deeply committed to providing access to new therapies for people living
with rare diseases such as NPC. There are currently no approved products to treat NPC in the US and we are pleased to make arimoclomol, a potential
first-in-class innovative oral therapy, available pre-commercially to US
patients through this program. We planto file for approval of arimoclomol in the US in H1 2020.