Trehalose - Bioblast Pharma

Drug Profile

Trehalose - Bioblast Pharma

Alternative Names: Cabaletta; Trehalose dihydrate

Latest Information Update: 01 Mar 2018

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At a glance

  • Originator Bioblast Pharma
  • Class Disaccharides; Glucans; Small molecules
  • Mechanism of Action Autophagy stimulants; Protein aggregation inhibitors
  • Orphan Drug Status

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

    Yes - Spinocerebellar degeneration; Oculopharyngeal muscular dystrophy
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Phase III Oculopharyngeal muscular dystrophy
  • Phase II Spinocerebellar degeneration
  • Discontinued Spinal muscular atrophy

Most Recent Events

  • 24 Jun 2018 Biomarkers information updated
  • 01 Dec 2017 Bioblast Pharma completes a phase III trial in Oculopharyngeal muscular dystrophy (In the elderly, In adults) in Israel (IV) (NCT02328482)
  • 11 Sep 2017 Discontinued - Preclinical for Spinal muscular atrophy in Israel (IV)
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