Orphazyme Stock Gains After FDA Update On Arimoclomol In Rare Neurodegenerative Disorder

  • Orphazyme A/S ORPH shares are moving higher after providing a regulatory update for arimoclomol in Niemann-Pick disease type C (NPC).
  • The Company had a collaborative dialogue with the FDA during the Type A meeting to discuss the Complete Response Letter.
  • Related: FDA Turned Down Arimoclomol Application Asking For Additional Evidence.
  • The FDA recommended that Orphazyme submit additional data, information, and analyses to address the CRL and engage in further interactions with the FDA to identify a pathway to resubmission.
  • The agency also agreed with the Company's proposal to remove the cognition domain from the NPCCSS endpoint. The primary endpoint is permitted to be recalculated using the 4-domain NPCCSS, subject to the submission of the additional requested information.
  • The FDA affirmed requiring additional pharmacodynamic (PD) / pharmacokinetics (PK) data to bolster the confirmatory evidence already submitted.
  • Related Link: Investors React Positively To Data From Orphazyme's Neurodegenerative Disease Candidate.
  • An EMA's opinion from the Committee for Medicinal Products for Human Use (CHMP)on arimoclomol in NPC is expected in Q1 2022.
  • Related content: Benzinga's Full FDA Calendar.
  • Price Action: ORPH shares are up 13.6% at $4.42 during the premarket session on the last check Monday.
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