Clinical data and new research from Sanofi’s rare blood disorders portfolio of marketed and investigational therapies for people with hemophilia, immune thrombocytopenia (ITP) and acquired thrombotic thrombocytopenic purpura (aTTP) will be presented at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress, July 17-21, 2021.
“Breaking barriers for people living with rare blood disorders requires that we push ourselves to transform the therapeutic landscape,” said Karin Knobe, MD, PhD, Head of Development, Rare Diseases and Rare Blood Disorders at Sanofi. “We deliver on that promise with our robust portfolio and pipeline of novel technologies that are designed to help address the underlying conditions or unmet needs for people with debilitating rare blood disorders. At ISTH, we are sharing data across all our innovative approaches and collectively, the data reinforce how our pipeline and portfolio may unlock new possibilities in the treatment of rare blood disorders.”
Breaking barriers in hemophilia: helping to evolve the treatment paradigm
Hemophilia is a rare, genetic bleeding disorder in which a person’s blood does not clot properly. Despite advances in treatment options in recent years, limitations still exist. Sanofi’s two marketed extended half-life factor replacement therapies shifted a two-decades-old treatment paradigm when launched in 2014. Today, with the molecules in its hemophilia pipeline, Sanofi is focused on delivering potential first-in-class and best-in-class therapies with the goal of bringing efficacy and convenience to a new level for patients.
Fitusiran is an investigational, subcutaneously administered small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors and has the potential to transform treatment as it would be the only prophylactic therapy with as few as six injections per year. Population pharmacokinetic and pharmacodynamic modeling data to characterize the antithrombin (AT) lowering dynamics in hemophilia patients treated with fitusiran will be shared in a poster presentation. These data support the revised fitusiran dose and dosing regimen implemented in the ongoing adult and adolescent studies. READ MORE
by Sanofi, Press Release
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