Orphazyme AS (OZYMF)

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told you yesterday to get out,,Orphazyme A/S : Asked by FDA for More Data on Genetic Disorder Drug; Revises 2021 Outlook; Shares Fall Pre-Bell
06/18/2021 | 05:15am EDT
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(MT Newswires) -- Orphazyme A/S (ORPH) said Friday the US Food and Drug Administration is asking for more data on the company's new drug application for arimoclomol, an investigational treatment for Niemann-Pick disease type C, a rare and progressive genetic disorder.

In a letter from the FDA, the regulator is seeking more qualitative and quantitative data to support the validity and interpretation of the five-domain NPC Clinical Severity Scale, which the company used in a phase 2/3 study to measure its primary endpoint of progression in disease severity.

The regulator is also asking for additional data beyond the phase 2/3 study to strengthen the confirmatory evidence and support the benefit-risk assessment of the new drug application.

In a statement, Orphazyme Chief Executive Christophe Bourdon said the company will focus on the European approval of the drug candidate while "assessing the potential path forward in the US." They expect an opinion from the European regulator's Committee for Medicinal Products for Human Use in Q4, with a potential market approval in the region by Q1 2022.

"In the short-term, we will need to reduce our costs substantially and freeze all company efforts not related to clinical and regulatory activities to support approval for [Niemann-Pick disease type C]," Bourdon said.

In connection with this, Orphazyme said it now expects 2021 operating expenses of 700 million Danish kroner ($112.1 million) to 800 million kroner, down from the previous range of 800 million kroner to 850 million kroner. Operating loss for the year is now pegged at 670 million kroner to 700 million kroner, compared with the previous outlook of a loss of 100 million kroner to 150 million kroner, according to the statement.

Cash position at the end of the year is now projected to be about 50 million kroner, down from the prior outlook of 350 million kroner.

Orphazyme stock fell more than 55% ahead of Friday's market open.

Price: 14.56, Change: -1.65, Percent Change: -10.18


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