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Company announcement No. 16/2021 Inside information Copenhagen - June 18 , 2021 - Orphazyme A / S ( ORPHA.CO ; ORPH ) , a late - stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases , today announced it has received a Complete Response Letter ( CRL ) from the U.S. Food and Drug Administration ( FDA ) 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 ( NPC ) . ORPHA ZYME Orphazyme provides regulatory update from FDA on arimoclomol for Niemann - Pick disease type C The FDA issued the CRL based on needing additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5 - domain NPC Clinical Severity Scale ( NPCCSS ) and , in particular , the swallow domain . Further , the FDA noted in the CRL that additional data are needed to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit - risk assessment of the NDA . Orphazyme A / S Ole Maaløes Vej 3 DK - 2200 Copenhagen N 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 . www.orphazyme.com Company Registration No. 32266355 " 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 , " said CEO Christophe Bourdon . He continued : " We will focus our efforts on pursuing the European regulatory approval , with CHMP opinion expected in Q4 2021 and potential Marketing Authorization in Q1 2022. We are assessing the potential path forward in the U.S. in partnership with the FDA . 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 NPC . " " 1 Deputy Chairman of the Board of Directors , Bo Jesper Hansen , said : " As representative for Orphazyme's shareholders and as a shareholder myself , I am extremely disappointed . I strongly believe there is a path forward for Orphazyme based on our pursuit of regulatory approval from the European Medicines Agency and continued dialogue with the FDA . Meeting these milestones in NPC will take great sacrifice from everyone in the organization , while we as a Board assist the management team in protecting as much value as possible . " Changed outlook for 2021 As stated in Orphazyme's Annual Report 2020 , 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 our commercial efforts and our development activities . The outcome of the FDA decision has significant influence on Orphazyme's outlook for full - year 2021 . The conference calls can be accessed by dialing : United Kingdom , local Denmark , Copenhagen France , Paris Netherlands , Amsterdam Sweden , Stockholm ● Orphazyme now expects operating expenses of DKK 700-720 million ( previously DKK 800-850 million ) resulting in an expected operating loss of DKK 670-700 million ( previously DKK 100-150 million ) . Cash position at year end 2021 is expected to be approx . DKK 50 million ( previously DKK 350 million ) . Orphazyme will provide an update and further information in the coming weeks . ● Conference calls and webcasts Orphazyme will host two conference calls / webcasts on June 18 , 2021 at 8.00 AM CET and 2.00 PM CET ( 8.00 AM EDT ) to accommodate European and U.S. time zones . The same information will be provided at both conference calls . +44 ( 0 ) 8444819752 +45 32720417 +33 ( 0 ) 170700781 +31 ( 0 ) 207956614 +46 ( 0 ) 856618467 Page 1 of 2