cerliponase alfa (brineura) Report issue

Biologics Recombinant protein Orphan Drug FDA Approved FDA First in Class Priority Review FDA

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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

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

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