Development of novel treatment options for patients with haemophilia

Summary

Treatment of haemophilia has vastly improved over the last years, but many needs are still unmet. Baxter is continuously pursuing the aim to provide new therapeutic options to patients with haemophilia and to their treating physicians. In fact, there are several opportunities to improve existing therapies, e.g., by new indications for existing products, the introduction of new products, and by novel therapeutic approaches other than factor replacement. Among these, Baxter is working on a number of innovations, such as pharmacokinetics-tailored factor VIII prophylaxis, bypassing agent prophylaxis with FEIBA in inhibitor patients, development of a longer acting pegylated recombinant FVIII, a new recombinant factor IX, a new recombinant factor FVIIa, the first recombinant von Willebrand factor, recombinant ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) as well as gene therapy to cure haemophilia B. Conclusion: 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.

Zusammenfassung

Die Behandlung der Hämophilie hat sich in den letzten Jahren wesentlich weiterentwickelt, dennoch besteht großer Bedarf zu weiteren Verbesserungen. Baxter verfolgt energisch das Ziel, neue Therapiemöglichkeiten für Patienten mit Hämophilie und ihre behandelnden Ärzte zu bieten. Tatsächlich gibt es eine Reihe von Möglichkeiten, Therapien zu verbessern, z. B. durch neue Indikationen für bestehende Produkte, Einführung neuer Produkte und innovative Therapieansätze alternativ zum Faktorersatz. Unter anderem arbeitet Baxter an Innovationen, wie z. B. der Pharmakokinetik-gesteuerten Faktor-VIII-Prophylaxe, der Prophylaxe mit „Bypassingagents” (FEIBA) bei Inhibitor-Patienten, einem lang wirkenden pegylierten rekombinanten Faktor FVIII, einem neuen rekombinanten Faktor IX, einem neuen rekombinanten Faktor VIIa, dem ersten rekombinanten von-Willebrand-Faktor, einer rekombinanten ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) und der Gentherapie für Hämophilie B. Schlussfolgerung: Baxter hat sich dem Patientenwohl verpflichtet und arbeitet an einer mehr und mehr individualisierten Behandlung, an der Effizienzsteigerung von aktuellen Produkten, an der Entwicklung neuer Produkte und an neuen vorteilhafteren Therapieansätzen.

Keywords

Haemophilia - prophylaxis - long-acting factor VIII - gene therapy

Schlüsselwörter

Hämophilie - Prophylaxe - lang wirkender Faktor VIII - Gentherapie

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