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Vosoritide - BioMarin Pharmaceutical

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Drug Profile

Vosoritide - BioMarin Pharmaceutical

Alternative Names: BMN-111; Modified C-natriuretic peptide - BioMarin Pharmaceutical; Modified recombinant human C-type natriuretic peptide - BioMarin Pharmaceutical; ProCNP38; VOXZOGO

Latest Information Update: 22 Aug 2025

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At a glance

  • Originator BioMarin Pharmaceutical
  • Class Cyclic peptides; Natriuretic agents; Natriuretic peptides; Recombinant proteins
  • Mechanism of Action Atrial natriuretic factor receptor B agonists
  • Orphan Drug Status

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

    Yes - Achondroplasia; Hypochondroplasia
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Achondroplasia
  • Phase III Hypochondroplasia
  • Phase II Noonan syndrome; Short stature; Turner's syndrome

Most Recent Events

  • 30 Jul 2025 Phase-II clinical trials in Hypochondroplasia (In children, In infants, In neonates) (SC, Injection) in USA (NCT07126262)
  • 20 Jun 2025 Phase-III clinical trials in Hypochondroplasia (In children, In adults) in USA, Australia, France, Canada, Germany, Italy, Japan, Spain and United Kingdom (SC) (NCT07073014)
  • 01 May 2025 BioMarin announces intention to launch Vosoritide for Hypochondroplasia in 2027

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