Orphazyme finds path forward for Niemann-Pick therapy in subgroup data

Orphazyme A/S (CSE:ORPHA) said arimoclomol significantly reduced disease progression in a pair of predefined subgroups in a Phase II/III trial to treat Niemann-Pick disease type C (NPC). The company plans to meet with FDA and EMA mid-year to discuss an approval pathway, with regulatory applications planned in 1H20 for the small molecule inducer of heat shock protein 70 (Hsp70).

In September, Orphazyme said arimoclomol missed the trial's primary endpoints in the overall patient population of reducing disease progression as measured by the 5-domain NPC Clinical Severity Scale (NPC-CSS) (p=0.0506) and of improving Clinical Global Impression of Improvement (CGI-I) from baseline to 12 months (p=1.00) vs. placebo (see "Orphazyme Seeking Path Forward for Arimoclomol in NPC After Phase II/III Miss")...