Drug Profile


Alternative Names: Cabaletta; Trehalose dihydrate

Latest Information Update: 12 Jun 2017

Price : $50

At a glance

  • Originator Bioblast Pharma
  • Class Disaccharides; Glucans; Small molecules
  • Mechanism of Action 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
  • Preclinical Spinal muscular atrophy

Most Recent Events

  • 05 Jun 2017 Trehalose is available for licensing as of 12 Jun 2017.
  • 05 Jun 2017 Bioblast Pharma has patent protection for use of trehalose in treatment of Huntington's disease in USA
  • 05 Jun 2017 Health Canada approves clinical trial application for phase IIb trial of trehalose in Oculopharyngeal muscular dystrophy
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