Peringatan Keamanan

There is no information on the LD50 of eteplirsen. There is no experience with overdose of eteplirsen.L40015

Eteplirsen

DB06014

biotech approved investigational

Deskripsi

Eteplirsen is a synthetic antisense oligonucleotide and a phosphorodiamidate morpholino oligomer. It consists of a six-membered morpholino ring replacing the five-membered ribofuranosyl rings found in natural DNA and RNA.L40015

Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and premature death most commonly due to respiratory or cardiac complications.A244930 It is caused by loss-of-function mutations in the DMD gene coding for dystrophin, an essential protein involved in maintaining the structural integrity and function of muscle fibres. Eteplirsen was first approved by the FDA in September 2016 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene, which codes for dystrophin, that is amenable to exon 51 skipping. Eteplirsen directly works on the DMD gene to promote dystrophin production. Eteplirsen was the first treatment for DMD approved by the FDA.A244925

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) The elimination half-life (t1/2) of eteplirsen was three to four hours.[L40015]
Volume Distribusi Following weekly intravenous infusion of eteplirsen at 30 mg/kg, the mean apparent volume of distribution (Vss) was 600 mL/kg. No accumulation is seen with once-weekly dosing.[L40015] Eteplirsen is not widely distributed.[A244925]
Klirens (Clearance) The total clearance of eteplirsen was 339 mL/hr/kg following 12 weeks of therapy with 30 mg/kg/week.[L40015]

Absorpsi

Following single or multiple intravenous infusions, the peak plasma concentrations (Cmax) of eteplirsen occurred near the end of infusion (i.e., 1.1 to 1.2 hours across a dose range of 0.5 mg/kg/week to 50 mg/kg/week). The inter-subject variability for eteplirsen Cmax and AUC range from 20 to 55%.L40015

Metabolisme

Eteplirsen does not undergo hepatic metabolism.L40015 As with other phosphorodiamidate morpholino oligomers, it is not favorable to metabolism.A244925

Rute Eliminasi

Renal clearance of eteplirsen accounts for approximately 67-70% of the administered dose within 24 hours of intravenous administration.A244925,L40015

Interaksi Obat

0 Data
Tidak ada data.

Target Protein

DMD-001 gene (exon 51 target site)

Referensi & Sumber

Artikel (PubMed)
  • PMID: 23907995
    Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM: Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10.
  • PMID: 28280301
    Lim KR, Maruyama R, Yokota T: Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545. doi: 10.2147/DDDT.S97635. eCollection 2017.
  • PMID: 30856119
    Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM: Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019;6(2):213-225. doi: 10.3233/JND-180351.

Contoh Produk & Brand

Produk: 2 • International brands: 0
Produk
  • Exondys 51
    Injection • 50 mg/1mL • Intravenous • US • Approved
  • Exondys 51
    Injection • 50 mg/1mL • Intravenous • US • Approved

Sekuens Gen/Protein (FASTA)

Sekuens dimuat saat dibutuhkan agar halaman tetap ringan.
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