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

Global Availability (8 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus

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Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Cerliponase alfa
Generic Namecerliponase alfa
Brands1 brand available
Active Ingredientcerliponase alfa
Drug Classneuronal ceroid lipofuscinosis type 2
ManufacturerBioMarin Pharmaceutical
Dosage Formsintracerebroventricular infusion, 150mg/5mL
Medical CodeA16AB17
Orphan StatusYes — Orphan Drug
Cold ChainRequired
Lead Time30 days
Reg. StatusFDA Approved, EMA Approved
Clinical TrialNCT01907087
Countries8 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes