Apadamtase alfa

DB15164

biotech approved investigational

Deskripsi

Thrombotic thrombocytopenic purpura (TTP) is a rare blood clotting disorder characterized by thrombocytopenia, microangiopathic hemolytic anemia, and various degrees of organ damage.A262076 It may be inherited (congenital TTP; cTTP) or acquired due to autoantibodies (immune-mediated TTP; iTTP), although cTTP accounts for <5% of all cases of TTP.A262076 Patients with cTTP have a severe deficiency of a plasma metalloproteinase called ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13), which is responsible for cleaving large von Willebrand factor multimers and preventing the formation of microthrombi - this deficiency in ADAMTS13 is responsible for the microangiopathic complications associated with cTTP.A262076 Standard therapy for cTTP involves prophylactic plasma infusions to supplement insufficient ADAMTS13 plasma levels.A262071

Apadamtase alfa, sold under the brand name Adzynma (ADAMTS13, recombinant-krhn), was approved by the FDA in November 2023 for use as an enzyme replacement therapy in patients with cTTP.L48756

Struktur Molekul 2D

Struktur tidak tersedia

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

Profil Farmakokinetik

Waktu Paruh (Half-Life) Following the intravenous administration of 40 IU/kg of rADAMTS13, the mean half-life (t<sub>1/2</sub>) is 47.8 hours.[L48746] The mean residence time (MRT<sub>0-inf</sub>) is 63.8 hours.[L48746]
Volume Distribusi -
Klirens (Clearance) -

Absorpsi

The average (Cave) and maximum (Cmax) serum concentrations of rADAMTS13 following intravenous administration of 40 IU/kg were 0.30 IU/mL and 1.15 IU/mL, respectively.L48746 The AUC0-inf and AUC0-168h were 57.6 IU.h/mL and 57.2 IU.h/mL, respectively.L48746

Metabolisme

As with other therapeutic proteins, the rADAMTS13 is likely degraded via catabolic processes to smaller peptides and amino acids.

Rute Eliminasi

Data eliminasi belum tersedia.

Interaksi Obat

0 Data
Tidak ada data.

Target Protein

von Willebrand factor VWF

Referensi & Sumber

Artikel (PubMed)
  • PMID: 32914526
    Zheng XL, Vesely SK, Cataland SR, Coppo P, Geldziler B, Iorio A, Matsumoto M, Mustafa RA, Pai M, Rock G, Russell L, Tarawneh R, Valdes J, Peyvandi F: ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020 Oct;18(10):2496-2502. doi: 10.1111/jth.15010. Epub 2020 Sep 11.
  • PMID: 32914582
    Zheng XL, Vesely SK, Cataland SR, Coppo P, Geldziler B, Iorio A, Matsumoto M, Mustafa RA, Pai M, Rock G, Russell L, Tarawneh R, Valdes J, Peyvandi F: ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020 Oct;18(10):2486-2495. doi: 10.1111/jth.15006. Epub 2020 Sep 11.

Contoh Produk & Brand

Produk: 2 • International brands: 0
Produk
  • Adzynma
    Injection, powder, lyophilized, for solution; Kit • 500 [iU]/1[iU] • Intravenous • US • Approved
  • Adzynma
    Injection, powder, lyophilized, for solution; Kit • 1500 [iU]/1[iU] • Intravenous • US • Approved

Sekuens Gen/Protein (FASTA)

Sekuens dimuat saat dibutuhkan agar halaman tetap ringan.
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