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Company announcement No. 21/2021 Orphazyme A / S Ole Maaløes Vej 3 DK - 2200 Copenhagen N CVR No .: 32266355 Copenhagen , Denmark , August 31 , 2021 - Orphazyme A / S ( ORPHA.CO ; ORPH ) ( " the Company " ) , a late - stage biopharmaceutical company , today announces its Interim Report First Half 2021 for the period January 1 - June 30 , 2021 . ● Interim Report First Half 2021 " We remain steadfast in our belief that arimoclomol holds significant potential for people living with NPC and we remain committed to pursuing a path to regulatory approval in Europe and the United States , " said Christophe Bourdon , Chief Executive Officer of Orphazyme . " While maintaining our patients on the Early Access Programs across key countries , we have executed on our restructuring plan , enabling significant cost savings to the company and are pursuing approval for arimoclomol . In the fourth quarter of 2021 , we expect a CHMP opinion from EMA on arimoclomol and expect to provide further detail on our path forward in the US following the conclusion of a Type A meeting with the FDA . We are assessing different possibilities for obtaining additional funding to sustain operations in 2022 and beyond " . Pipeline Updates First Half 2021 Announced top - line results from both the Phase 2/3 trial of arimoclomol in Inclusion Body Myositis and the Phase 3 trial of arimoclomol in Amyotrophic Lateral Sclerosis ; neither trial met its primary endpoint and we have ceased further development of arimoclomol in these indications Received a Complete Response Letter from FDA following its review of the new drug application for arimoclomol in NPC ; we are assessing the path forward with FDA Marketing authorization application for arimoclomol for NPC remains underway with the European Medicines Agency and a CHMP opinion is expected during Q4 2021 Our Early Access Programs ( EAP ) for arimoclomol in the United States , Germany and France continued through the first half of 2021 , with approx . 100 patients participating in the EAP as of June 30 , 2021 ● ORPHA ZYME ● Presented 12 - month and 24 - month results from the open - label extension of the Phase 2/3 trial of arimoclomol in NPC at the 17th Annual WORLDSymposium Scientific meeting and the Parseghian Scientific Conference for NPC Research respectively . The results demonstrate that arimoclomol provided a sustained benefit to study participants through 36 months and are consistent with the previously reported safety profile Financial and Business Highlights First Half 2021 Appointed Christophe Bourdon as Chief Executive Officer , effective April 1 , 2021 Announced company restructuring that will result in a significant headcount reduction ( ~ 70 remaining FTEs expected by year - end ) , substantial cost savings , and a focus on activities to support potential approval of arimoclomol in Europe and in the US For the first six months of 2021 , Orphazyme reported a net loss of DKK 463.8.0 million or DKK 13.27 per share ( basic and diluted ) compared to a net loss of DKK 251.4 million or DKK 9.77 per share ( basic and diluted ) for the same period in 2020 Beginning in 2021 and for the six - month period ended June 30 , 2021 , Orphazyme recognized net revenue of DKK 13.2 million from the sale of arimoclomol for treatment of NPC under the ATU ( remunerated early access program ) in France Page 1 of 28