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Orphazyme's Arimoclomol Disappoints For Muscle-Wasting Disorder

Pivotal Trial Failure For ‘Pipeline-In-A-Product’

Executive Summary

The Danish biotech's stock sank after arimoclomol failed in a Phase II/III trial for inclusion body myositis. Orphazyme is hoping for much better news in the coming weeks as the US FDA makes its decision on the drug as a treatment for Niemann-Pick disease Type C.

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The EMA's medicines evaluation committee has told Orphazyme it is unlikely to recommend approval of the rare disease drug arimoclomol when it meets at the end of March, prompting the Danish firm to assess its strategic options.

Uncertain Future For Orphazyme After EMA Snub

The EMA's medicines evaluation committee has told Orphazyme it will not approve the rare disease drug arimoclomol when it meets at the end of March, prompting the Danish firm to assess its strategic options.

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