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Adrabetadex - Mandos/National Institutes of Health

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Drug Profile

Adrabetadex - Mandos/National Institutes of Health

Alternative Names: 2-Hydroxypropyl-B-Cyclodextrin - Mandos; 2-Hydroxypropyl-beta-cyclodextrin - Mandos; HP-beta-CD - Mandos; HPBCD - Mandos; Hydroxypropyl betadex - Mandos; Hydroxypropyl betadex intrathecal - Mandos; Intrathecal hydroxypropyl betadex - Mandos; IT-HPBCD - Mandos; VTS 270

Latest Information Update: 26 Feb 2026

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At a glance

  • Originator National Institutes of Health (USA)
  • Developer Mallinckrodt plc; Mandos; National Institute of Child Health and Human Development; Washington University School of Medicine
  • Class Antidementias; Beta-Cyclodextrins; Ethers
  • Mechanism of Action Binding agents; Cholesterol modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Niemann-Pick disease type C
  • New Molecular Entity No

Highest Development Phases

  • Preregistration Niemann-Pick disease type C

Most Recent Events

  • 23 Feb 2026 Adrabetadex receives priority review status for Neimann-Pick disease type C in USA
  • 23 Feb 2026 US FDA assigns PDUFA action date of (17/08/2026) for Adrabetadex for Niemann-Pick disease type C
  • 23 Feb 2026 US FDA accepts NDA for Adrabetadex for Niemann-Pick disease type C for review

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