03.01.20
• 12-month data from open-label extension trial show sustained effect in reducing disease progression over two years
• New genetic sub-group analysis recommended by US Food and Drug Administration supports
robustness of results
• Filling of New Drug Application in US on track for H1 2020, with expected approval in H1 2021
19.12.19
Data show that Trappsol® Cyclo™, the Company’s proprietary hydroxypropyl beta cyclodextrin drug, reduces levels of trapped cholesterol in liver tissue of Niemann-Pick Disease Type C Patients...
Read more29.11.19
We are joining 140+ organisations in a call to all political parties to put children and young people at the heart of this General Election...
Read more19.11.19
Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”
Read more31.10.19
Publication describes favorable safety profile and efficacy in neurologic and other disease features in individual NPC patients following intravenous administration of drug...
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