Cerliponase Alfa — Membership Pricing

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About Cerliponase Alfa

Cerliponase alfa, marketed as Brineura, is a highly specialized enzyme replacement therapy crucial for treating Neuronal Ceroid Lipofuscinosis Type 2 (CLN2 disease), a rare and severe neurodegenerative condition. Due to its unique nature and administration, this vital medication often comes with significant costs. TheDrugClub is committed to helping our members explore potential savings and access options for their prescription needs, including complex therapies like cerliponase alfa.

Cerliponase alfa, known commercially as Brineura, represents a significant advancement in the treatment of Neuronal Ceroid Lipofuscinosis Type 2 (CLN2 disease), also recognized as a form of Batten disease. CLN2 disease is a devastating, progressive neurodegenerative disorder primarily affecting children, leading to developmental regression, seizures, and loss of motor and cognitive function. Without treatment, the disease is fatal, typically in late childhood.

How Cerliponase Alfa Works

CLN2 disease is caused by a deficiency in the tripeptidyl peptidase 1 (

The information above is for educational purposes only and does not constitute medical advice. Consult your healthcare provider before making medication decisions.

What Cerliponase Alfa is approved for (FDA label)

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. ( 1 )

Source: Read the full FDA label on DailyMed. Label effective date: August 5, 2024.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Boxed warning for Cerliponase Alfa (FDA label)

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. Initiate BRINEURA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue BRINEURA and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ]. WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) Initiate BRINEURA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue BRINEURA and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )

Source: FDA prescribing information (DailyMed). Set ID: 485ab3c5-0359-4878-872f-2fe1e7df4047. SPL document ID: 1ebeb049-90ec-42ff-9361-b6fa38d79515. Label effective date: August 5, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Dosage forms and strengths for Cerliponase Alfa (FDA label)

Injection: BRINEURA 150 mg/5 mL (30 mg/mL) solution, two single-dose vials per carton co-packaged with Intraventricular Electrolytes Injection 5 mL in a single-dose vial. BRINEURA is a clear to slightly opalescent and colorless to pale yellow solution. Intraventricular Electrolytes is a clear to colorless solution [see How Supplied/Storage and Handling (16) ] . Injection: BRINEURA 150 mg/5 mL (30 mg/mL) solution, two single-dose vials per carton co-packaged with Intraventricular Electrolytes Injection 5 mL in a single-dose vial. ( 3 )

Source: FDA prescribing information (DailyMed). Set ID: 485ab3c5-0359-4878-872f-2fe1e7df4047. SPL document ID: 1ebeb049-90ec-42ff-9361-b6fa38d79515. Label effective date: August 5, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Adverse reactions for Cerliponase Alfa (FDA label)

The following adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Meningitis and Other Intraventricular Access Device-Related Infections [see Warnings and Precautions (5.2) ] Intraventricular Access Device-Related Complications [see Warnings and Precautions (5.3) ] Cardiovascular Adverse Reactions [see Warnings and Precautions (5.4) ] Infusion-Associated Reactions [see Warnings and Precautions (5.5) ] 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. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact BioMarin at 1-866-906-6100 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Source: FDA prescribing information (DailyMed). Set ID: 485ab3c5-0359-4878-872f-2fe1e7df4047. SPL document ID: 1ebeb049-90ec-42ff-9361-b6fa38d79515. Label effective date: August 5, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Contraindications for Cerliponase Alfa (FDA label)

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) [see Warnings and Precautions (5.2) ] . any acute intraventricular access device-related complication (e.g., leakage, extravasation of fluid, or device failure) [see Warnings and Precautions (5.3) ]. 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). ( 4 ) Any acute intraventricular access device-related complication (e.g., leakage, extravasation of fluid, or device failure). ( 4 ) Patients with ventriculoperitoneal shunts. ( 4 )

Source: FDA prescribing information (DailyMed). Set ID: 485ab3c5-0359-4878-872f-2fe1e7df4047. SPL document ID: 1ebeb049-90ec-42ff-9361-b6fa38d79515. Label effective date: August 5, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Warnings and precautions for Cerliponase Alfa (FDA label)

Meningitis and Other Intraventricular Access Device-Related Infections : Monitor the device insertion site for signs of infection. ( 4 , 5.2 ) 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. ( 4 , 5.3 ) Cardiovascular Adverse Reactions : Monitor vital signs before, during, and post-infusion. Monitor Electrocardiogram (ECG) in patients with a history of bradycardia, conduction disorder, or with structural heart disease, during the infusion.

Source: FDA prescribing information (DailyMed). Set ID: 485ab3c5-0359-4878-872f-2fe1e7df4047. SPL document ID: 1ebeb049-90ec-42ff-9361-b6fa38d79515. Label effective date: August 5, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.