Cerliponase alfa
Pharmaceutical drug / From Wikipedia, the free encyclopedia
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.[3] 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.[4]
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Trade names | Brineura |
AHFS/Drugs.com | Monograph |
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Routes of administration | Intraventricular |
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Formula | C2657H4042N734O793S11 |
Molar mass | 59308.57 g·mol−1 |
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[5]