Orphazyme Halves Premarket As FDA Rejects Genetic Disorder Candidate

By Dhirendra Tripathi – Shares of Orphazyme (NASDAQ:) looked set for an even bigger fall Friday after an over 10% decline in the previous session as the Food and Drug Administration rejected the company’s experimental treatment, arimoclomol, for a neurodegenerative rare disease known as Niemann-Pick disease type C.

The shares were down more than 52% in premarket trading as the company seeks to reduce costs substantially and “freeze all company efforts not related to clinical and regulatory activities to support approval for NPC.”

With the Danish company’s initial outlook dependent on the regulatory decision, it has now been forced to lower that.

Orphazyme now expects operating loss of DKK 670 million to DKK 700 million (approximately $107 million to $112 million) for the full year.

It previously estimated the operating loss to come in the range of DKK 100 million to DKK150 million (approximately $16 million to $24 million).

Niemann-Pick disease type C mostly appears in childhood. The incidence of the disease is estimated to be 1 in 100,000 live births and it is estimated that there are ~1,800 patients across U.S. and Europe, according to Orphazyme’s 2020 annual report.

In more aggressive forms, NPC is frequently fatal by the time patients reach their 20s.


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