Development of novel treatment options for patients with haemophilia

Ehrlich, Hartmut J.; Wong, Wing Yen; Ewenstein, Bruce M.; Dockal, Michael; Turecek, P. L.; Gringeri, A.; Chehadeh, H.; Löw-Baselli, A.; Scheiflinger, Friedrich; Reininger, Armin J.

doi:10.1055/s-0037-1619792

Cited by 7 publications

(3 citation statements)

References 14 publications

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“…Several new recombinant clotting factor concentrates with unchanged pharmacokinetic characteristics have entered the market recently or will do so within the forthcoming year. These include a rFXIII A subunit concentrate from Novo Nordisk , a rFVIII concentrate (N8) from Novo Nordisk , a rFVIII concentrate from Octapharma (Octapharma AG, Lachen, Switzerland) , a rFIX concentrate from Baxter , a rVWF concentrate from Baxter and a porcine rFVIII which has been initially developed by Inspiration and now overtaken by Baxter .…”

Section: New Recombinant Clotting Factor Concentrates With Unchanged mentioning

confidence: 99%

Novel products for haemostasis – current status

Oldenburg¹,

Albert²

2014

Haemophilia

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Summary Currently, new clotting factor concentrates are becoming available or are in advanced clinical studies that will significantly improve the treatment of patients with Haemophilia A or Haemophilia B. Various technologies are applied to extend half‐life and/or allow for alternative routes of administration, e.g. subcutaneous route. Today, the advances for recombinant factor IX are significantly with half‐life extensions to up to 100 h, allowing substitution intervals of 1–2 weeks. For recombinant factor VIII (FVIII) products the effect so far is only moderate, as the half‐life extension is limited to about 15–18 h by the clearance of FVIII through its binding to von Willebrand factor. However, novel products applying new technologies with significantly extended half‐life are already at the horizont, as a bispecific antibody that mimics FVIII. The pharmacokinetic improvements of the new products will lead to a revision of our current treatment regimens, with regard to intended trough levels, number of tolerated bleeds and likely will drive a greater individualization of regimens. Clearly, the potential of anti drug antibody response for these modified proteins must not be higher than with our current products. Another challenge are the increasingly diverse biochemical characteristics of the new products, that have to be considered when determining potencies and also when monitoring treatment in patients with the various available assays. Despite these challenges, the new products will significantly improve treatment and quality of life for our patients with haemophilia.

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Section: New Recombinant Clotting Factor Concentrates With Unchanged mentioning

confidence: 99%

Novel products for haemostasis – current status

Oldenburg¹,

Albert²

2014

Haemophilia

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“…Sklonowanie genu czynnika krzepnięcia IX (FIX, factor IX) w 1982 roku i genu czynnika krzepnięcia VIII (FVIII, factor VIII) w 1984 roku umożliwiło opracowanie technologii produkcji rekombinowanych czynników krzepnięcia, których istotną przewagą nad osoczopochodnymi czynnikami krzepnięcia jest niższe ryzyko przeniesienia cząstek zakaźnych [1,2]. Ale przewaga leków rekombinowanych nie ogranicza się do większego bezpieczeństwa ich stosowania.…”

Section: Wprowadzenieunclassified

Postępy w leczeniu chorych na hemofilie

Windyga

2015

Hematologia

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“…Several further PEGylated products are currently in clinical trials, e.g., blood clotting factors such as PEGylated rFVIII (BAX855; Baxter) (Ehrlich et al 2013 ), developed using a proprietary PEGylation technology (Nektar Therapeutics), and PEGylated B-domain-deleted recombinant factor VIII (BAY94-9027; Bayer) (Ivens et al 2013 ), as well as several PEGylated versions of human growth hormone (hGH), including NNC126-0083 (Novo Nordisk) (de Schepper et al 2011 ), ARX201 (Ambrx) and ACP001 (Ascendis). However, despite the success of PEGylation technology so far, drawbacks have emerged and the pharmaceutical industry has started to search for PEG alternatives.…”

Section: Pegylation and Chemical Coupling To Other Polymersmentioning

confidence: 99%

Biobetters

2015

AAPS Advances in the Pharmaceutical Sciences Series

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Development of novel treatment options for patients with haemophilia

Abstract: Baxter is truly committed to the benefit for the patient, and therefore engaged in providing a more and more individualized treatment, in increasing efficiency of current products, in developing new products and new approaches with added value.

Cited by 7 publications

References 14 publications

Novel products for haemostasis – current status

Novel products for haemostasis – current status

Postępy w leczeniu chorych na hemofilie

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