Peringatan Keamanan

There is no information available regarding the acute toxicity (LD50) and overdose of cerliponase alfa.

Cerliponase alfa

DB13173

biotech approved investigational

Deskripsi

Cerliponase alfa is a hydrolytic lysosomal N-terminal tripeptidyl peptidase-1 (TPP1).L51319 On April 27, 2017, cerliponase alfa was first approved by the FDA as the first treatment for neuronal ceroid lipofuscinosis type 2 (CLN2), also known as TPP1 deficiency.L51339 It was also approved by the EMA on May 30, 2017 L51324 and by Health Canada on December 19, 2018.L51329 CLN2 is a predominantly pediatric-onset neurodegenerative disease caused by a deficiency in the lysosomal enzyme TPP1, leading to progressive impairment in motor and cognitive function.A264354, A264369 As a recombinant human TPP1, cerliponase alfa is used as an enzyme replacement therapy to restore the levels of TPP1 in patients with CLN2.L51339

Struktur Molekul 2D

Struktur tidak tersedia

Peta Jejaring Molekuler
Legenda: Obat • Target • Gen • Enzim • (Panah → menunjukkan arah efek / relasi) • Transporter • Carrier

Profil Farmakokinetik

Waktu Paruh (Half-Life) Following intraventricular infusions of 300 mg of cerliponase alfa, the median CSF half-life was 6.2, 7.4 and 7.7 hours on day 1, week 5 and week 13, respectively.[A264354, L51319]
Volume Distribusi Following intraventricular infusions of 300 mg of cerliponase alfa, the median CSF volume of distribution at steady state was 245, 196 and 186 ml on day 1, week 5 and week 13, respectively.[A264354, L51319]
Klirens (Clearance) Following intraventricular infusions of 300 mg of cerliponase alfa, the median CSF clearance was 32.3, 24.2 and 28.7 ml/h on day 1, week 5 and week 13, respectively.[A264354, L51319]

Absorpsi

Cerliponase alfa pharmacokinetics have high inter-subject and intra-subject variability.L51319 Following intraventricular infusions of 300 mg of cerliponase alfa, the median maximum concentration (Cmax) in cerebrospinal fluid (CSF) was 1260, 1630 and 1390 ug/ml at day 1, week 5, and week 13, respectively. The median Tmax was 4.5, 4.3 and 4.3 h on day 1, week 5 and week 13, respectively. The area under the CSF concentration–time curve from 0 to the last measurable concentration (AUC0–t) 9290, 12,400 and 10,500 ug x h/ml, respectively.A264354, L51319

Metabolisme

Cerliponase alfa is a protein and is expected to be degraded through peptide hydrolysis.L51319

Rute Eliminasi

Data eliminasi belum tersedia.

Interaksi Obat

0 Data
Tidak ada data.

Target Protein

Cation-independent mannose-6-phosphate receptor IGF2R

Referensi & Sumber

Artikel (PubMed)
  • PMID: 28589525
    Markham A: Cerliponase Alfa: First Global Approval. Drugs. 2017 Jul;77(11):1247-1249. doi: 10.1007/s40265-017-0771-8.
  • PMID: 31884868
    Lewis G, Morrill AM, Conway-Allen SL, Kim B: Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2. J Child Neurol. 2020 Apr;35(5):348-353. doi: 10.1177/0883073819895694. Epub 2019 Dec 29.
  • PMID: 33882967
    Mole SE, Schulz A, Badoe E, Berkovic SF, de Los Reyes EC, Dulz S, Gissen P, Guelbert N, Lourenco CM, Mason HL, Mink JW, Murphy N, Nickel M, Olaya JE, Scarpa M, Scheffer IE, Simonati A, Specchio N, Von Lobbecke I, Wang RY, Williams RE: Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients. Orphanet J Rare Dis. 2021 Apr 21;16(1):185. doi: 10.1186/s13023-021-01813-5.

Contoh Produk & Brand

Produk: 3 • International brands: 0
Produk
  • Brineura
    Injection, solution; Kit • 150 mg/5mL • Intraventricular • US • Approved
  • Brineura
    Solution • 150 mg / 5 mL • Intracerebral • Canada • Approved
  • Brineura
    Injection, solution • 150 mg • Intracerebral • EU • Approved

Sekuens Gen/Protein (FASTA)

Sekuens dimuat saat dibutuhkan agar halaman tetap ringan.
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