Drug Profile


Alternative Names: Cabaletta; Trehalose dihydrate

Latest Information Update: 31 Jan 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 - Oculopharyngeal muscular dystrophy; Spinocerebellar degeneration
  • New Molecular Entity No

Highest Development Phases

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

Most Recent Events

  • 18 Jan 2017 Bioblast Pharma has patent protection for use of trehalose in treatment of Spinocerebellar degeneration in USA
  • 18 Jan 2017 Efficacy and adverse event data from a phase II trial in Spinocerebellar degeneration released by Bioblast Pharma
  • 01 Nov 2016 Bioblast Pharma completes a phase-II clinical trial in Spinocerebellar degeneration in Israel (IV) (NCT02147886)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top