Peringatan Keamanan

Toxicity information regarding efanesoctocog alfa is not readily available. Patients experiencing an overdose are at an increased risk of severe adverse effects such as hypersensitivity, including anaphylaxis.L45379 Symptomatic and supportive measures are recommended. The carcinogenicity, mutagenicity or effects on fertility of efanesoctocog alfa have not been evaluated.L45379

Efanesoctocog alfa

DB16662

biotech approved investigational

Deskripsi

Efanesoctocog alfa (BIVV001) is a recombinant factor VIII (FVIII) analogue fusion protein used for the routine prophylaxis, perioperative management of bleeding and on-demand treatment and control of bleeding episodes in patients with hemophilia A.L45379 The use of FVIII replacement products is beneficial in patients with hemophilia A; however, their quality of life can be affected due to frequent doses.A257464 Efanesoctocog alfa was designed to have an extended half-life, and to surpass the half-life ceiling to which other forms of recombinant FVIII are subjected due to their association with von Willebrand factor (VWF). Endogenous VWF protects FVIII from degradation but also sets a half-life of approximately 15 to 19 h. To extend the half-life of FVIII, efanesoctocog alfa is fused to dimeric Fc, a D'D3 domain of VWF, and two XTEN polypeptides.A257464,A257469

In February 2023, efanesoctocog alfa was approved by the FDA as a new class of factor VIII therapy for hemophilia A.L45379,L45414

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) Efanesoctocog alfa administered as a single intravenous injection of 50 IU/kg had a terminal half-life of 39.9 h in children between 1 and 6 years old, 42.4 h in children between 6 and 12 years old, 44.6 h in adolescents between 12 and 18 years old, and 48.2 h in adults.[L45379]
Volume Distribusi Efanesoctocog alfa administered as a single intravenous injection of 50 IU/kg had a volume of distribution at steady-state of 38.0 mL/kg in children between 1 and 6 years old, 38.1 mL/kg in children between 6 and 12 years old, 34.9 mL/kg in adolescents between 12 and 18 years old, and 31.0 mL/kg in adults.[L45379]
Klirens (Clearance) Efanesoctocog alfa administered as a single intravenous injection of 50 IU/kg had a clearance of 0.74 mL/h/kg in children between 1 and 6 years old, 0.681 mL/h/kg in children between 6 and 12 years old, 0.582 mL/h/kg in adolescents between 12 and 18 years old, and 0.493 mL/h/kg in adults.[L45379]

Absorpsi

Efanesoctocog alfa administered as a single intravenous injection of 50 IU/kg had an AUC of 6710 IU?h/dL in children between 1 and 6 years old, 7190 IU?h/dL in children between 6 and 12 years old, 8350 IU?h/dL in adolescents between 12 and 18 years old, and 9850 IU?h/dL in adults. In all groups, a single dose of 50 IU/kg of efanesoctocog alfa led to high sustained FVIII activity and a prolonged half-life. As age increased, there was a tendency of higher AUC values; however, this effect was not clinically significant. Sex, race (White, Asian), VWF antigen activity, hematocrit level, blood type, HCV status, or HIV status did not have a clinically significant effect on efanesoctocog alfa either. At steady state (week 26), the pharmacokinetic profile of efanesoctocog alfa was comparable to the one obtained after the first dose.L45379

Metabolisme

As a fusion protein, efanesoctocog alfa is expected to be metabolized by proteases throughout the body.

Rute Eliminasi

Data eliminasi belum tersedia.

Interaksi Obat

0 Data
Tidak ada data.

Target Protein

Coagulation factor IX F9
Coagulation factor X F10
Low-density lipoprotein receptor-related protein 2 LRP2
Basement membrane-specific heparan sulfate proteoglycan core protein HSPG2

Referensi & Sumber

Artikel (PubMed)
  • PMID: 32905674
    Konkle BA, Shapiro AD, Quon DV, Staber JM, Kulkarni R, Ragni MV, Chhabra ES, Poloskey S, Rice K, Katragadda S, Fruebis J, Benson CC: BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A. N Engl J Med. 2020 Sep 10;383(11):1018-1027. doi: 10.1056/NEJMoa2002699.
  • PMID: 32078672
    Seth Chhabra E, Liu T, Kulman J, Patarroyo-White S, Yang B, Lu Q, Drager D, Moore N, Liu J, Holthaus AM, Sommer JM, Ismail A, Rabinovich D, Liu Z, van der Flier A, Goodman A, Furcht C, Tie M, Carlage T, Mauldin R, Dobrowsky TM, Liu Z, Mercury O, Zhu L, Mei B, Schellenberger V, Jiang H, Pierce GF, Salas J, Peters R: BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice. Blood. 2020 Apr 23;135(17):1484-1496. doi: 10.1182/blood.2019001292.
  • PMID: 35466511
    Demers M, Aleman MM, Kistanova E, Peters R, Salas J, Seth Chhabra E: Efanesoctocog alfa elicits functional clot formation that is indistinguishable to that of recombinant factor VIII. J Thromb Haemost. 2022 Jul;20(7):1674-1683. doi: 10.1111/jth.15741. Epub 2022 May 22.
  • PMID: 36720133
    von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K: Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. N Engl J Med. 2023 Jan 26;388(4):310-318. doi: 10.1056/NEJMoa2209226.
  • PMID: 34794179
    Lissitchkov T, Willemze A, Katragadda S, Rice K, Poloskey S, Benson C: Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study. Blood Adv. 2022 Feb 22;6(4):1089-1094. doi: 10.1182/bloodadvances.2021006119.

Contoh Produk & Brand

Produk: 6 • International brands: 1
Produk
  • Altuviiio
    Kit; Powder, for solution • 250 [iU]/3mL • Intravenous • US • Approved
  • Altuviiio
    Kit; Powder, for solution • 500 [iU]/3mL • Intravenous • US • Approved
  • Altuviiio
    Kit; Powder, for solution • 1000 [iU]/3mL • Intravenous • US • Approved
  • Altuviiio
    Kit; Powder, for solution • 2000 [iU]/3mL • Intravenous • US • Approved
  • Altuviiio
    Kit; Powder, for solution • 3000 [iU]/3mL • Intravenous • US • Approved
  • Altuviiio
    Kit; Powder, for solution • 4000 [iU]/3mL • Intravenous • US • Approved
International Brands
  • Altuviiio — Bioverativ Therapeutics

Sekuens Gen/Protein (FASTA)

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