Pharma Focus Asia

Orphazyme Receives Orphan Drug Designation To Arimoclomol For Inclusion Body Myositis From The U.S. FDA

Wednesday, November 08, 2017

Orphazyme A/S, a Danish biotech company with a late stage orphan drug pipeline, today announced that the U.S. Food and Drug Administration ("FDA"), Office of Orphan Products Development, has granted an orphan drug designation to arimoclomol for the treatment of the orphan disease Inclusion Body Myositis ("IBM"). As described in the offering circular published by Orphazyme A/S on 6 November 2017, the Company applied to the FDA for the designation in September 2017.

The Company focuses, among others, on sporadic inclusion body myositis ("sIBM") and has already obtained an EU orphan drug designation for sIBM from the European Medicines Agency ("EMA"), which was granted in 2016.

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