Expert opinion on investigational drugs · 2024
Factor IX stimulants in preclinical and early phase trials for hemophilia B treatment.
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Franchini Massimo, Focosi Daniele
Аннотация
INTRODUCTION: Hemophilia B is a X-linked rare inherited bleeding disorder characterized by coagulation factor IX (FIX) deficiency. Therapy for hemophilia B is aimed at replacing the FIX deficiency by means of several plasma-derived or recombinant FIX products. The recent availability of recombinant FIX concentrates with a prolonged FIX half-life represented a great technological advance, permitting more spaced drug infusions and reducing treatment burden among hemophilia B patients.
AREAS COVERED: This review summarizes the main preclinical and phase 1/2 studies investigating the innovative hemostatic products for hemophilia B replacement therapy.
EXPERT OPINION: The significant recent technological advantages in the treatment of hemophilia B has led to the development of innovative FIX products aimed at further extending FIX half-life and using increasingly effective and convenient modes of administration. These novel hemostatic agents, currently in the preclinical or early clinical phase of development, carry the potential of improving patients' health status and quality of life. Continuous research is anyway needed to offer such patients a concrete chance of conducting a normal existence, like to non-affected age-matched individuals.
AREAS COVERED: This review summarizes the main preclinical and phase 1/2 studies investigating the innovative hemostatic products for hemophilia B replacement therapy.
EXPERT OPINION: The significant recent technological advantages in the treatment of hemophilia B has led to the development of innovative FIX products aimed at further extending FIX half-life and using increasingly effective and convenient modes of administration. These novel hemostatic agents, currently in the preclinical or early clinical phase of development, carry the potential of improving patients' health status and quality of life. Continuous research is anyway needed to offer such patients a concrete chance of conducting a normal existence, like to non-affected age-matched individuals.