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BBDF 101

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Drug Profile

BBDF 101

Alternative Names: Batten-1; BBDF-101

Latest Information Update: 27 Jun 2025

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At a glance

  • Originator Beyond Batten Disease Foundation
  • Developer Beyond Batten Disease Foundation; Theranexus
  • Class
  • Mechanism of Action Alpha-glucosidase inhibitors; Autophagy stimulants; Glucosylceramide synthase inhibitors; Protein aggregation inhibitors
  • Orphan Drug Status

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

    Yes - Neuronal ceroid lipofuscinosis
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Neuronal ceroid lipofuscinosis

Most Recent Events

  • 30 May 2025 Beyond Batten Disease Foundation completes a phase I/II trial in Neuronal ceroid lipofuscinosis in USA (PO) (NCT05174039)
  • 09 May 2023 Theranexus and Beyond Batten Disease Foundation plans a phase III trial for Neuronal ceroid lipofuscinosis in USA and Europe (PO, Capsule)
  • 02 Mar 2021 Beyond Batten Disease Foundation (BBDF) enters into an agreement with Actelion Pharmaceuticals to provide Miglustat for the development of BBDF 101 for Batten disease .

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