What kind of drug is seripalase (BRINEURA)? Detailed explanation of main indications and clinical applications

Seripase (BRINEURA, common name: cerliponase alfa) is a recombinant human brain lysosomal enzyme and is an enzyme replacement therapy drug. It slows neurodegenerative damage by replenishing missing or malfunctioning enzymes and helping to break down abnormal accumulations of material within cells. BRINEURA is mainly developed for specific genetic neurometabolic diseases and is currently one of the few approved treatments for this type of disease.

BRINEURAis mainly used for the treatment of **CLN2 type gangliosidosis (Late-infantile neuronal ceroid lipofuscinosis, LINCL)**. The disease is a rare hereditary neurodegenerative disease caused by mutations in the TPP1 gene leading to a deficiency of the TTP1 enzyme in the body, causing the accumulation of abnormal substances in the brain, eventually leading to the progressive decline of motor function, language ability and cognitive function. BRINEURA’s enzyme replacement therapy can delay the progression of symptoms and improve the quality of life of children.

BRINEURAAdministered through intracerebroventricular injection (intracerebroventricular infusion), active enzymes are directly delivered into the cerebrospinal fluid, thus overcoming blood-brain barrier restrictions and allowing the enzymes to reach nerve cells. Clinical studies have shown that regular dosing can delay the loss of motor and speech skills in children with CLN2 type 2. Drugs need to be administered in professional medical institutions and managed by experienced neurology teams to ensure safety and efficacy.

BRINEURAIn clinical use, the efficacy is mainly evaluated through motor ability and language scores, and potential adverse reactions need to be monitored, including fever, headache, injection site infection and allergic reactions. Parents or guardians need to closely cooperate with the medical team for follow-up and conduct neurological function and imaging examinations before and after treatment. Although BRINEURA cannot completely cure CLN2 gangliosidosis, it can significantly delay the progression of the disease, providing patients with a valuable therapeutic window and the opportunity to improve their quality of life.

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