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Rhttp1 (CERLIPONASE ALFA)

BioMarin · FDA-approved approved Enzyme Quality 50/100

Rhttp1 (generic name: CERLIPONASE ALFA) is a Hydrolytic Lysosomal N-terminal Tripeptidyl Peptidase Enzyme drug developed by BioMarin. It is currently FDA-approved (first approved 2017) for Late-infantile neuronal ceroid lipofuscinosis.

Cerliponase alfa works by inhibiting the breakdown of a protein called tripeptidyl peptidase, which is deficient in patients with late-infantile neuronal ceroid lipofuscinosis.

Cerliponase alfa, also known as Relyvio, is a small molecule drug developed by Biogen (previously by Biogen and now by Biogen's competitor) and currently owned by Biogen. It is a hydrolytic lysosomal N-terminal tripeptidyl peptidase inhibitor, approved by the FDA in 2017 for the treatment of late-infantile neuronal ceroid lipofuscinosis. This rare genetic disorder affects the brain and causes progressive vision loss, seizures, and loss of motor skills. Cerliponase alfa is administered intravenously and is not yet available as a generic version. Key safety considerations include the risk of infusion-related reactions and the potential for immune system activation.

At a glance

Generic nameCERLIPONASE ALFA
SponsorBioMarin
Drug classHydrolytic Lysosomal N-terminal Tripeptidyl Peptidase
ModalityEnzyme
Therapeutic areaNeuroscience
PhaseFDA-approved
First approval2017

Mechanism of action

Imagine your brain has a recycling system that breaks down old or damaged proteins. In people with this disorder, this system doesn't work properly, leading to a buildup of toxic waste. Cerliponase alfa helps by blocking the enzyme that breaks down a specific protein, allowing the brain to recycle it more efficiently.

Approved indications

Boxed warnings

Common side effects

Serious adverse events

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

SourceUsed for
FDA labelMechanism, indications, dosing, boxed warnings, drug interactions
ClinicalTrials.govTrial enrolment, design, endpoints, results

Competitive intelligence

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Frequently asked questions about Rhttp1

What is Rhttp1?

Rhttp1 (CERLIPONASE ALFA) is a Hydrolytic Lysosomal N-terminal Tripeptidyl Peptidase drug developed by BioMarin, indicated for Late-infantile neuronal ceroid lipofuscinosis.

How does Rhttp1 work?

Cerliponase alfa works by inhibiting the breakdown of a protein called tripeptidyl peptidase, which is deficient in patients with late-infantile neuronal ceroid lipofuscinosis.

What is Rhttp1 used for?

Rhttp1 is indicated for Late-infantile neuronal ceroid lipofuscinosis.

Who makes Rhttp1?

Rhttp1 is developed and marketed by BioMarin (see full BioMarin pipeline at /company/biomarin).

What is the generic name of Rhttp1?

CERLIPONASE ALFA is the generic (nonproprietary) name of Rhttp1.

What drug class is Rhttp1 in?

Rhttp1 belongs to the Hydrolytic Lysosomal N-terminal Tripeptidyl Peptidase class. See all Hydrolytic Lysosomal N-terminal Tripeptidyl Peptidase drugs at /class/hydrolytic-lysosomal-n-terminal-tripeptidyl-peptidase.

When was Rhttp1 approved?

Rhttp1 was first approved on 2017.

What development phase is Rhttp1 in?

Rhttp1 is FDA-approved (marketed).

What are the side effects of Rhttp1?

Common side effects of Rhttp1 include Seizures, Device-related complications, Hypersensitivity, Pyrexia, Hematoma, Pleocytosis. Serious adverse events: Intraventricular access device-related CNS infections, Hypoxia, Device leakage, Low mean arterial pressure.

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