Drug Profile


Alternative Names: Cabaletta; Trehalose dihydrate

Latest Information Update: 30 Nov 2016

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 - Oculopharyngeal muscular dystrophy; Spinocerebellar degeneration
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    Oculopharyngeal muscular dystrophy
  • New Molecular Entity No

Highest Development Phases

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

Most Recent Events

  • 01 Nov 2016 Bioblast Pharma completes a phase-II clinical trial in Spinocerebellar degeneration in Israel (IV) (NCT02147886)
  • 24 Oct 2016 Efficacy, pharmacokinetic and adverse events data from a phase I pharmacokinetic trial in Oculopharyngeal muscular dystrophy released by BioBlast Pharma
  • 12 Sep 2016 Adverse events data from a phase II/III (HOPEMD) trial in Oculopharyngeal muscular dystrophy released by BioBlast Pharma
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