News

Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease

Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease type C

Copenhagen, Denmark, March 22, 2022 – Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme” or the “Company”), a late-stage biopharmaceutical company, announces that, following the receipt of the negative Trend Vote by the Committee for Medicinal Products for Human Use (CHMP) as announced on February 23, 2022...

Orphazyme Announces Update on Regulatory Review of Arimoclomol in the European Union

IB1001-301: Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study

Orphazyme provides update for planned NDA resubmission for arimoclomol for the treatment of Niemann-Pick disease type C in the United States