cerliponase alfa (Rx)

Brand and Other Names:Brineura, recombinant human tripeptidyl peptidase 1 (rhtpp1)
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Dosing & Uses

AdultPediatric

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Dosage Forms & Strengths

injection for intraventricular administration kit

  • Cerliponase alfa: 150mg/5mL (2 vials)
  • Intraventricular electrolytes: 5mL (1 vial)
  • Also includes administration kit (see Administration)

CLN2 Disease

Indicated to slow the loss of ambulation in symptomatic pediatric patients aged ≥3 yr with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, a form of Batten disease

<3 years: Safety and efficacy not established

≥3 years: 300 mg by intraventricular infusion at rate of 2.5 mL/hr once every other week

Follow with intraventricular infusion of electrolytes at rate of 2.5 mL/hr

The total infusion time is ~4.5 hr for cerliponase alfa plus the electrolytes

Also see Administration

Pretreatment

  • Pretreatment with antihistamines with or without antipyretics or corticosteroids is recommended 30-60 minutes before intraventricular infusion
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Adverse Effects

>10%

Pyrexia (71%)

ECG abnormalities (71%)

Decreased CSF protein (71%)

Vomiting (63%)

Seizures (50%)

Hypersensitivity (46%)

Increased CSF protein (21%)

Hematoma (21%)

Headache (17%)

Irritability (17%)

Pleocytosis (17%)

1-10%

Device-related infection (8%)

Bradycardia (8%)

Feeling jittery (8%)

Hypotension (8%)

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Warnings

Contraindications

Acute intraventricular access device-related complications (eg, leakage, device failure, device-related infection)

Patients with ventriculoperitoneal shunts

Cautions

Must be administered using aseptic technique to reduce the risk of infection; healthcare professionals should inspect the scalp for skin integrity to ensure the intraventricular access device is not compromised prior to each infusion (see Administration)

Monitor vital signs before infusion starts, periodically during infusion, and postinfusion in a healthcare setting

Perform ECG monitoring during infusion in patients with a history of bradycardia, conduction disorder, or with structural heart disease, as some patients with CLN2 disease may develop conduction disorders or heart disease; in patients without cardiac abnormalities, regular 12-lead ECG evaluations should be performed every 6 months

Hypersensitivity reactions reported during the infusion or within 24 hr of completion; observe patient during and after infusion; inform caregivers of signs and symptoms of anaphylaxis and instruct them to seek immediate medical care if these occur

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Pregnancy

Pregnancy

There are no available data on use in pregnant women to inform a drug-associated risk of pregnancy-related outcomes

Lactation

Unknown if distributed in human breast milk

Pregnancy Categories

A:Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

B:May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

C:Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

D:Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

X:Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

NA:Information not available.

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Pharmacology

Mechanism of Action

Recombinant form of human tripeptidyl peptidase (TPP-1) that provides enzyme replacement therapy; this results in a restored breakdown of the lysosomal storage materials that cause CLN2 disease

CLN2 late infantile disease is caused by a deficiency in the TPP-1 enzyme, a lysosomal enzyme that cleaves peptides into amino acids; TPP-1 deficiency causes abnormal storage of proteins and lipids in neurons and other cells, resulting in impaired cellular function

Pharmacokinetics

Expected to be degraded through peptide hydrolysis

CSF

  • Tmax: 4.3-4.5 hr
  • Cmax: 1260-1630 mcg/mL
  • AUC: 9290-12,400 mcg·hr/mL
  • Vd: 186-245 mL
  • Distribution half-life: 2.5-7.7 hr

Plasma

  • Tmax: 12-12.3 hr
  • Cmax: 1-1.9 mcg/mL
  • AUC: 9.5-40.1 mcg·hr/mL
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Administration

Intraventricular Preparation

Aseptic technique must be strictly observed during preparation and administration

Thaw cerliponase alfa and intraventricular electrolytes injection vials at room temperature for ~60 minutes

Do not thaw or warm vials any other way

Do not shake vials

Condensation will occur during thawing period

Do not refreeze vials or freeze syringes containing the drug or electrolyte solutions

Cerliponase alfa is a clear to slightly opalescent and colorless to pale yellow solution

Intraventricular electrolytes is a clear to colorless solution

Do not use if the solutions are discolored or if there is other foreign particulate matter in the solutions

Cerliponase alfa vials may occasionally contain thin translucent fibers or opaque particles; these naturally occurring particles are cerliponase alfa and are removed via the 0.2 micron inline filter without having a detectable effect on the purity or potency

Intraventricular electrolytes may contain particles, which appear during the thaw period; however, these dissolve when the solution reaches room temperature

Do not dilute cerliponase alfa or mix with any other drug

Administration kit includes

  • Two 20-mL syringes
  • Two syringe needles (21 ga, 25.4 mm)
  • One extension line
  • One infusion set with 0.2 micron inline filter
  • One port needle (22 ga, 16 mm)

Intraventricular Administration

Administered by, or under the direction of, a physician knowledgeable in intraventricular administration

Administered into the CSF by infusion via a surgically implanted reservoir and catheter (intraventricular access device); intended to be administered via the Codman HOLTER RICKHAM Reservoirs (Part Numbers: 82-1625, 82-1621, 82-1616) with the Codman Ventricular Catheter (Part Number: 82-1650)

The intraventricular access device must be implanted prior to the first infusion

It is recommended that the first dose be administered at least 5-7 days after device implantation

Each infusion consist of 10 mL cerliponase alfa followed by 2 mL of intraventricular electrolytes using an infusion set with a 0.2 micron line filter (provided in kit)

Infusion rate: cerlionase alfa 2.5 mL/hr and electrolytes 2.5 mL/hr; total infusion time is ~4.5 hr

The intraventricular electrolytes are used to flush the infusion line, port needle, and intraventricular access device in order to fully administer the cerliponase alfa dose and to maintain patency of the intraventricular access device

See prescribing information for diagram of intraventricular infusion procedure

Administer by B Braun Perfusor Space Infusion Pump System

  • The essential performance requirement for this syringe pump used to deliver cerliponase alfa are as follows
    • Delivery rate of 2.5 mL/hr with accuracy of +/- 1 mL/hr
    • Compatible with 20-mL syringes provided in the administration kit
    • Occlusion alarm setting to ≤281 mmHg

Storage

Store cerliponase alfa injection and intraventricular electrolytes injection upright in freezer (-25°C to -15°C) in original carton to protect from light

Administration kit: Store in original carton separately from cerliponase alfa (do not freeze)

Thawed product

  • Use thawed cerliponase alfa and intraventricular electrolytes immediately
  • If not used immediately, store unopened vials in the refrigerator at 2-8°C and use within 24 hr

Thawed product in syringes

  • Use product held in labeled syringes immediately
  • If not used immediately, store product held in labeled syringes in the refrigerator at 2-8°C up to 4 hr prior to infusion
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Formulary

FormularyPatient Discounts

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The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

Tier Description
1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
NC NOT COVERED – Drugs that are not covered by the plan.
Code Definition
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