Branaplam - Novartis

Drug Profile

Branaplam - Novartis

Alternative Names: LMI 070; NVS-SM1

Latest Information Update: 09 Sep 2017

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At a glance

  • Originator Novartis
  • Class Cyclohexanes; 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.

  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Spinal muscular atrophy

Most Recent Events

  • 08 Sep 2017 Chemical structure information added
  • 05 Sep 2017 Phase-I/II development is ongoing in Spinal muscular atrophy (In infants) in Belgium, Denmark, Germany and Italy (NCT02268552)
  • 27 Oct 2014 Preclinical trials in Spinal muscular atrophy in Switzerland (PO)
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