Uncertain Future For Orphazyme After EMA Snub
Agency Set To Reject Arimoclomol For Niemann-Picks Type C
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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The sponsors of two investigational drugs that have previously experienced key regulatory setbacks are set to give oral explanations to the European Medicines Agency about why they believe their products should be approved in the EU.
Following Orphazyme’s disappointment in the US regarding its investigational drug for an ultrarare disease, the company has said it will need more time to address outstanding issues raised by EU reviewers about the same medicine.
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.