cerliponase alfa
Orphan Drug Cold Chain RequiredFDA Approved, EMA Approved
Description
Cerliponase alfa is a recombinant human tripeptidyl peptidase 1 (TPP1) enzyme replacement therapy administered via intracerebroventricular infusion. It is specifically approved for neuronal ceroid lipofuscinosis type 2 (CLN2) and represents the first enzyme replacement therapy to cross the blood-brain barrier for lysosomal storage disorders. While approved for CLN2, it has been studied in other NCL subtypes including CLN11.
Indications & Therapeutic Use
neuronal ceroid lipofuscinosis type 2, Batten disease
Linked Diseases:
Global Availability (8 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
cerliponase alfa
| Generic Name | cerliponase alfa |
| Brands | 1 brand available |
| Active Ingredient | cerliponase alfa |
| Drug Class | neuronal ceroid lipofuscinosis type 2 |
| Manufacturer | BioMarin Pharmaceutical |
| Dosage Forms | intracerebroventricular infusion, 150mg/5mL |
| Medical Code | A16AB17 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 30 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT01907087 |
| Countries | 8 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes