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Branaplam - Novartis

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Drug Profile

Branaplam - Novartis

Alternative Names: Branaplam hydrochloride - Novartis; LMI 070; NVP-LMI070-AAA; NVP-LMI070-NX; NVS-SM1

Latest Information Update: 28 May 2025

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At a glance

  • Originator Novartis
  • Class Cyclohexanes; Piperidines; Pyrazoles; Pyridazines; Small molecules
  • Mechanism of Action Survival of motor neuron 2 protein modulators
  • Orphan Drug Status

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

    Yes - Spinal muscular atrophy; Huntington's disease
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Spinal muscular atrophy
  • No development reported Unspecified
  • Discontinued Huntington's disease

Most Recent Events

  • 28 May 2025 No recent reports of development identified for phase-I development in Spinal-muscular-atrophy(In volunteers) in United Kingdom (PO)
  • 28 May 2025 No recent reports of development identified for phase-I development in Unspecified(In volunteers) in United Kingdom (PO)
  • 11 Nov 2024 Discontinued - Phase-II for Huntington's disease (In the elderly, In adults) in Lithuania (PO) (NCT05111249) (EudraCT2020-000105-92)

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