cerliponase alfa (brineura) Report issue

Biologics Recombinant protein Orphan Drug FDA Approved FDA First in Class
AACT ChEMBL DrugBank Drugs@FDA

Active Ingredient History

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Cerliponase alfa, marketed as Brineura, is an enzyme replacement treatment for Batten disease, a neurodegenerative lysosomal storage disease. Specifically, Cerliponase alfa is meant to slow loss of motor function in symptomatic children over three years old with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The disease is also known as tripeptidyl peptidase-1 (TPP1) deficiency, a soluble lysosomal enzyme deficiency. Approved by the United States Food and Drug Administration (FDA) on 27 April 2017, this is the first treatment for a neuronal ceroid lipofuscinosis of its kind, acting to slow disease progression rather than palliatively treat symptoms by giving patients the TPP1 enzyme they are lacking.   Wikipedia

Chemistry

More Chemistry

Pharmacology

  • Mechanisms of Action:
  • Multi-specific: No
  • Black Box: No
  • Availability: Prescription Only
  • Delivery Methods: Parenteral
  • Pro Drug: No

Alternative names

bmn190 | bmn 190 | bmn-190 | brineura | cerliponase alfa | cerliponase alpha | immature cell growth-inhibiting gene 1 protein | immature human tripeptidyl-peptidase 1 | immature lysosomal pepstatin-insensitive protease | immature tripeptidyl-peptidase i

Organizations (2)

Research & Development (2)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (1)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (3)

Approved Indications (1)


 

Neuronal Ceroid-Lipofuscinoses

Experimental Indications - Clinical Trial Phases 1-4 (3)


 

Genetic Diseases, Inborn (Phase 2)

Infant (Phase 2)

Neuronal Ceroid-Lipofuscinoses ()

Overview

  • Highest Phase: Phase 2
  • # of Trials: 5
  • # of Trial Organizations: 2

Trials by Phase

Trial Phase Start Date Organizations Indications

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