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Cerliponase alfa - BioMarin Pharmaceutical

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Drug Profile

Cerliponase alfa - BioMarin Pharmaceutical

Alternative Names: BMN 190; BRINEURA; Brineura; recombinant human tripeptidyl peptidase-1; rhTPP1

Latest Information Update: 26 Jul 2024

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At a glance

  • Originator BioMarin Pharmaceutical
  • Class Enzymes; Peptide hydrolases; Recombinant proteins
  • Mechanism of Action Tripeptidyl-peptidase 1 replacements
  • 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

  • Marketed Neuronal ceroid lipofuscinosis

Most Recent Events

  • 24 Jul 2024 US FDA appoved sBLA for treatment of patients with Neuronal ceroid lipofuscinosis type 2 (In neonates, In infants, In children) in USA
  • 24 Jul 2024 Efficacy and adverse events data from a phase II trial in Neuronal ceroid lipofuscinosis type 2 was released by BioMarin Pharmaceutical
  • 20 Apr 2022 BioMarin Pharmaceutical completes a phase II trial in Neuronal ceroid lipofuscinosis (In adolescents, In children) in USA, Germany, Italy and United Kingdom (Intracerebroventricular) (NCT02678689) (EudraCT2015-000891-85)

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