Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program
Simoctocog alfa (human-cl rhFVIII, Nuwiq®) is a 4th generation recombinant FVIII (rFVIII), without chemical modification or fusion with any other protein/fragment. Nuwiq® is produced in a human embryonic kidney cell line (HEK293F), which ensures human-specific post-translational protein processing....
| Main Authors: | , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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SAGE Publishing
2019-06-01
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| Series: | Therapeutic Advances in Hematology |
| Online Access: | https://doi.org/10.1177/2040620719858471 |
| _version_ | 1819008649403564032 |
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| author | Toshko Lissitchkov Anna Klukowska John Pasi Craig M. Kessler Robert Klamroth Raina J. Liesner Larisa Belyanskaya Olaf Walter Sigurd Knaub Johann Bichler Martina Jansen Johannes Oldenburg |
| author_facet | Toshko Lissitchkov Anna Klukowska John Pasi Craig M. Kessler Robert Klamroth Raina J. Liesner Larisa Belyanskaya Olaf Walter Sigurd Knaub Johann Bichler Martina Jansen Johannes Oldenburg |
| author_sort | Toshko Lissitchkov |
| collection | DOAJ |
| description | Simoctocog alfa (human-cl rhFVIII, Nuwiq®) is a 4th generation recombinant FVIII (rFVIII), without chemical modification or fusion with any other protein/fragment. Nuwiq® is produced in a human embryonic kidney cell line (HEK293F), which ensures human-specific post-translational protein processing. Nuwiq® was evaluated in seven prospective clinical studies in 201 adult and pediatric previously treated patients (PTPs) with severe hemophilia A. The NuProtect study in 110 previously untreated patients (PUPs) is ongoing. The mean half-life of Nuwiq® was 15.1–17.1 h in PTP studies with adults and adolescents, and 12.5 h in children aged 2–12 years. Clinical trials in PTPs demonstrated the efficacy and safety of Nuwiq® in the prevention and treatment of bleeds and as surgical prophylaxis. In the NuPreviq study of pharmacokinetic (PK)-guided personalized prophylaxis in 66 adult PTPs, 83% of patients had no spontaneous bleeds during 6 months of personalized prophylaxis and 57% were treated ⩽2 per week. No FVIII inhibitors were detected in PTPs after treatment with 43,267 injections and >80 million IU of Nuwiq®. Interim data for 66 PUPs with ⩾20 exposure days to Nuwiq® in NuProtect demonstrated a low cumulative high-titer inhibitor rate of 12.8% [actual incidence 12.1% (8/66)] and convincing efficacy and safety. |
| first_indexed | 2024-12-21T00:43:50Z |
| format | Article |
| id | doaj.art-7231c4278c1b4b3f877509384a9fb07f |
| institution | Directory Open Access Journal |
| issn | 2040-6215 |
| language | English |
| last_indexed | 2024-12-21T00:43:50Z |
| publishDate | 2019-06-01 |
| publisher | SAGE Publishing |
| record_format | Article |
| series | Therapeutic Advances in Hematology |
| spelling | doaj.art-7231c4278c1b4b3f877509384a9fb07f2022-12-21T19:21:35ZengSAGE PublishingTherapeutic Advances in Hematology2040-62152019-06-011010.1177/2040620719858471Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA programToshko LissitchkovAnna KlukowskaJohn PasiCraig M. KesslerRobert KlamrothRaina J. LiesnerLarisa BelyanskayaOlaf WalterSigurd KnaubJohann BichlerMartina JansenJohannes OldenburgSimoctocog alfa (human-cl rhFVIII, Nuwiq®) is a 4th generation recombinant FVIII (rFVIII), without chemical modification or fusion with any other protein/fragment. Nuwiq® is produced in a human embryonic kidney cell line (HEK293F), which ensures human-specific post-translational protein processing. Nuwiq® was evaluated in seven prospective clinical studies in 201 adult and pediatric previously treated patients (PTPs) with severe hemophilia A. The NuProtect study in 110 previously untreated patients (PUPs) is ongoing. The mean half-life of Nuwiq® was 15.1–17.1 h in PTP studies with adults and adolescents, and 12.5 h in children aged 2–12 years. Clinical trials in PTPs demonstrated the efficacy and safety of Nuwiq® in the prevention and treatment of bleeds and as surgical prophylaxis. In the NuPreviq study of pharmacokinetic (PK)-guided personalized prophylaxis in 66 adult PTPs, 83% of patients had no spontaneous bleeds during 6 months of personalized prophylaxis and 57% were treated ⩽2 per week. No FVIII inhibitors were detected in PTPs after treatment with 43,267 injections and >80 million IU of Nuwiq®. Interim data for 66 PUPs with ⩾20 exposure days to Nuwiq® in NuProtect demonstrated a low cumulative high-titer inhibitor rate of 12.8% [actual incidence 12.1% (8/66)] and convincing efficacy and safety.https://doi.org/10.1177/2040620719858471 |
| spellingShingle | Toshko Lissitchkov Anna Klukowska John Pasi Craig M. Kessler Robert Klamroth Raina J. Liesner Larisa Belyanskaya Olaf Walter Sigurd Knaub Johann Bichler Martina Jansen Johannes Oldenburg Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program Therapeutic Advances in Hematology |
| title | Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program |
| title_full | Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program |
| title_fullStr | Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program |
| title_full_unstemmed | Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program |
| title_short | Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program |
| title_sort | efficacy and safety of simoctocog alfa nuwiq r in patients with severe hemophilia a a review of clinical trial data from the gena program |
| url | https://doi.org/10.1177/2040620719858471 |
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