Orphazyme (ORPH) has received CRL from the FDA following its review of the new drug application for arimoclomol

June 18, 2021 6:09 AM EDT
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Orphazyme A/S (NASDAQ: 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).

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.

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.

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.

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 2021As 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.

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 webcastsOrphazyme 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.

The conference calls can be accessed by dialing:

  • United Kingdom, local +44 (0) 8444819752
  • Denmark, Copenhagen +45 32720417
  • France, Paris +33 (0) 170700781
  • Netherlands, Amsterdam +31 (0) 207956614
  • Sweden, Stockholm +46 (0) 856618467
  • Standard International +44 (0) 2071 928338
  • United States, New York +1 6467413167

Event Title: Orphazyme Investor Call Confirmation code for 8.00 AM CET: 2889481

Confirmation code for 2.00 PM CET (8.00 AM EDT): 9349928

Link for webcast at 8.00 AM CET: https://edge.media-server.com/mmc/p/q5yie99y

Link for webcast at 2.00 PM CET (8.00 AM EDT): https://edge.media-server.com/mmc/p/9k4qohms

An archive of the teleconferences/webcasts will be made available on Orphazyme's website, www.orphazyme.com, following the call.



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