Peringatan Keamanan

Miglustat has been administered at doses of up to 3000 mg/day (approximately 10 times the recommended starting dose administered to Gaucher patients) for up to six months in Human Immunodeficiency Virus (HIV)-positive patients. Adverse events observed in the HIV studies included granulocytopenia, dizziness, and paresthesia. Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or above.

Miglustat

DB00419

small molecule approved

Deskripsi

Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.

Struktur Molekul 2D

Berat 219.278
Wujud solid

Peta Jejaring Molekuler
Legenda: ObatTargetGenEnzim(Panah → menunjukkan arah efek / relasi)TransporterCarrier

Profil Farmakokinetik

Waktu Paruh (Half-Life) The effective half-life of miglustat is approximately 6 to 7 hours.
Volume Distribusi -
Klirens (Clearance) -

Absorpsi

Mean oral bioavailability is 97%.

Metabolisme

There is no evidence that miglustat is metabolized in humans.

Rute Eliminasi

Data eliminasi belum tersedia.

Interaksi Makanan

2 Data
  • 1. Take at the same time every day.
  • 2. Take with or without food.

Interaksi Obat

0 Data
Tidak ada data.

Target Protein

Ceramide glucosyltransferase UGCG

Referensi & Sumber

Artikel (PubMed)
  • PMID: 17720777
    van Giersbergen PL, Dingemanse J: Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol. 2007 Oct;47(10):1277-82. Epub 2007 Aug 24.
  • PMID: 16247743
    Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P: Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9.
  • PMID: 17689147
    Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE: Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol. 2007 Sep;6(9):765-72.
  • PMID: 12803929
    Moyses C: Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease. Philos Trans R Soc Lond B Biol Sci. 2003 May 29;358(1433):955-60.
  • PMID: 19956552
    Wraith JE, Imrie J: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat. Ther Clin Risk Manag. 2009;5:877-87. Epub 2009 Nov 18.
  • PMID: 14609352
    McCormack PL, Goa KL: Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6.
  • PMID: 14757169
    Andersson U, Reinkensmeier G, Butters TD, Dwek RA, Platt FM: Inhibition of glycogen breakdown by imino sugars in vitro and in vivo. Biochem Pharmacol. 2004 Feb 15;67(4):697-705.

Contoh Produk & Brand

Produk: 19 • International brands: 1
Produk
  • Miglustat
    Powder • 1 kg/1kg • - • US
  • Miglustat
    Capsule • 100 mg/1 • Oral • US • Generic • Approved
  • Miglustat
    Capsule • 100 mg/1 • Oral • US • Generic • Approved
  • Miglustat
    Capsule • 100 mg/1 • Oral • US • Generic • Approved
  • Miglustat
    Capsule • 100 mg/1 • Oral • US • Generic • Approved
  • Miglustat Dipharma
    Capsule • 100 mg • Oral • EU • Approved
  • Miglustat Dipharma
    Capsule • 100 mg • Oral • EU • Approved
  • Miglustat Dipharma
    Capsule • 100 mg • Oral • EU • Approved
Menampilkan 8 dari 19 produk.
International Brands
  • Zavesca — Actelion Pharmaceuticals

Sekuens Gen/Protein (FASTA)

Sekuens dimuat saat dibutuhkan agar halaman tetap ringan.
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