ORPH Orphazyme AS

By Colin Kellaher

Shares of Orphazyme A/S dropped more than 60% Friday after the U.S. Food and Drug Administration rejected the biopharmaceutical company's application for approval of arimoclomol for the treatment of the rare progressive genetic disorder Niemann-Pick disease type C, or NPC.

The Copenhagen company said it received a complete response letter from the FDA indicating that the agency wouldn't approve the application in its current form,

Orphazyme said the FDA requested more qualitative and quantitative evidence to substantiate the validity and interpretation of the five-domain NPC clinical severity scale, a disease-specific, clinician-reported outcome measure of disease severity and progression, particularly the swallow domain.

The company said the FDA also asked for more data to bolster confirmatory evidence beyond the single Phase 2/3 clinical trial to support the benefit-risk assessment of the application.

Orphazyme said it is assessing the potential path forward in the U.S. in partnership with the FDA, adding that it plans to cut costs substantially and freeze all efforts not related to clinical and regulatory activities to support approval for NPC.

The company said it will focus its efforts on pursuing European regulatory approval, with an opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use expected by the end of the year and potential marketing authorization in the first quarter of 2022.

Copenhagen-listed shares of Orphazyme were recently down 62% to 37.84 Danish krone ($6.08).

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

June 18, 2021 06:57 ET (10:57 GMT)

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