Orphazyme Gets CRL From FDA On Arimoclomol For Niemann-Pick Disease Type C; Updates FY21 Loss View

Danish rare disease company Orphazyme AS (ORPH) announced Friday that it has received a Complete Response Letter or CRL from the U.S. Food and Drug Administration following its review of the new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C or NPC.

Orphazyme further said the outcome of the FDA decision has significant influence on its outlook for full-year 2021.

For fiscal 2021, Orphazyme now expects operating loss of 670 million Danish kroner to 700 million kroner, compared to previously expected loss of 100 million kroner to 150 million kroner. Operating expenses are now expected to be 700 million kroner to 720 million kroner, down from previously expected 800 million kroner to 850 million million kroner.

The company earlier said that its initial outlook for the year was subject to various risks and uncertainties, including but not limited to the timing of regulatory decisions, the success of commercial efforts and development activities.

The CRL is issued for additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale or NPCCSS and, in particular, the swallow domain.

Further, the FDA requires additional data to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.

According to the company, a primary endpoint of the phase 2/3 clinical trial was progression in disease severity as measured by the 5-domain NPCCSS. This is a disease-specific measure of disease progression consisting of the five clinically most relevant domains to patients with NPC, caregivers and physicians.

CEO Christophe Bourdon said, "We are disheartened by the outcome of the FDA's review, given the urgent need for a new therapeutic option for NPC, but we remain committed to working with the regulators, with the goal of delivering arimoclomol to families managing this challenging disease."

The company said it will focus on pursuing the European regulatory approval, with CHMP opinion expected in the fourth quarter and potential Marketing Authorization in the first quarter next year.

In the short-term, the company said it will need to reduce costs substantially and freeze all company efforts not related to clinical and regulatory activities to support approval for NPC.

Cash position at year-end 2021 is expected to be around 50 million kroner, down from earlier projected 350 million kroner.

Orphazyme said it will provide an update and further information in the coming weeks.

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