Molecule
Cerliponase alfa is a prescription medicine indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. MedsAcross supplies Cerliponase alfa for export from India to licensed hospitals, importers and physicians acting for named patients, with the documents each country requires.
Information from the US FDA prescribing information, retrieved 12 November 2026. Not yet medically reviewed.How we review
From the regulator-approved product information. Each section names its source.
BRINEURA is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. BRINEURA is a hydrolytic lysosomal N-terminal tripeptidyl peptidase indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Source: US FDA prescribing information
CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. TPP1 has no known substrate specificity. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system (CNS), leading to progressive decline in motor function. Cerliponase alfa (rhTTP1), a proenzyme, is taken up by target cells in the CNS and is translocated to the lysosomes through the Cation Independent Mannose-6-Phosphate Receptor (CI-MPR, also known as M6P/IGF2 receptor). Cerliponase alfa is activated in the lysosome and the activated proteolytic form of rhTPP1 cleaves tripeptides from the N-terminus of proteins.
Source: US FDA prescribing information
Administration of BRINEURA should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. BRINEURA should be administered by or under the supervision of a physician experienced in intraventricular administration. Premedication of patients with antihistamines with or without antipyretics or corticosteroids is recommended. Prior to each infusion, inspect the scalp for signs of intraventricular access device reservoir leakage, failure, or potential infection. Recommended BRINEURA dosage is 300 mg administered once every other week as an intraventricular infusion. In patients less than 2 years of age, lower doses are recommended. Dosing is not recommended in patients less than 37 weeks post-menstrual age or those weighing less than 2.5 kg.
Source: US FDA prescribing information
BRINEURA is contraindicated in patients with: any sign or symptom of acute, unresolved localized infection on or around the device insertion site (e.g., cellulitis or abscess), or suspected or confirmed CNS infection (e.g., cloudy CSF or positive CSF gram stain, or meningitis). any acute intraventricular access device-related complication (e.g., leakage, extravasation of fluid, or device failure). ventriculoperitoneal shunts. Any sign or symptom of acute or unresolved localized infection on or around the device insertion site (e.g. cellulitis or abscess), or suspected or confirmed CNS infection (e.g., cloudy CSF or positive CSF gram stain, or meningitis).
Source: US FDA prescribing information
Boxed warning (hypersensitivity reactions including anaphylaxis): Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Meningitis and Other Intraventricular Access Device-Related Infections: Monitor the device insertion site for signs of infection. Intraventricular Access Device-Related Complications: Consult a neurosurgeon for any complications with the implanted device. In case of device-related complication, discontinue the infusion and refer to the device labeling for further instructions. Cardiovascular Adverse Reactions: Monitor vital signs before, during, and post-infusion.
Source: US FDA prescribing information
The following adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis Meningitis and Other Intraventricular Access Device-Related Infections Intraventricular Access Device-Related Complications Cardiovascular Adverse Reactions Infusion-Associated Reactions Most common adverse reactions (≥8%) are: pyrexia, ECG abnormalities, decreased CSF protein, vomiting, seizures, device-related complications, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infections, bradycardia, feeling jittery, and hypotension.
Source: US FDA prescribing information
Store upright in a freezer (-25°C to -15°C) in original carton to protect from light. Do not freeze. Administration Kit for use with BRINEURA: Store in original carton separately from BRINEURA.
Source: US FDA prescribing information
For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.
1 presentations of Cerliponase alfa, sorted by availability.
| Medicine | Manufacturer | Register | Availability |
|---|---|---|---|
| Brineura kit Cold chain | BioMarin Pharmaceutical | US FDA | Available on request |
1 brand names across 1 markets.
United States
Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.
Yes, on request. MedsAcross lists 1 presentations of Cerliponase alfa and sources them for licensed hospitals, importers and physicians against valid import documents.
The catalogue lists 1 brand names for Cerliponase alfa, including Brineura.
Yes. The US FDA first approved Cerliponase alfa on 27 April 2017, according to Drugs@FDA.
Yes. Cerliponase alfa has a central EU marketing authorisation (status: authorised), according to European Medicines Agency data.
At least one listed presentation of Cerliponase alfa must be kept cold, so it is shipped in temperature-controlled packaging. Check the storage line on each presentation.
Cerliponase alfa is classified under ATC code A16AB17 (Cerliponase alfa).
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