Michael Makris: I Hope The Bleeding Disorders Community Supports Kedrion Initiative for FV Deficiency
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at University of Sheffield, reposted from Kedrion Biopharma on LinkedIn:
”Factor V concentrate on the horizon!
I probably first heard about the possibility of a Factor V concentrate 20 years ago.
There was optimism, but then everything went quiet, and most of us had given up on Kedrion ever bringing a factor V concentrate to market.
I was so pleased to see this press release indicating that the project is up and running once again.
I really hope they succeed.
Factor V deficiency is the only inherited clotting factor deficiency with no good effective treatment.
Fresh frozen plasma (FFP) is not the long-term answer for those individuals with severe deficiency.
I hope the bleeding disorders community support this Kedrion initiative.”
Quoting Kedrion Biopharma‘s post:
”Behind every rare disease, there are people living with limited treatment options, often relying only on non-specific replacement therapies and supportive treatments.
Today, we share an important step forward.
Kedrion has received Orphan Drug Designation from EMA for its investigational therapy for Congenital Factor V Deficiency, an ultra-rare bleeding disorder.
For many patients, non-specific replacement therapies and supportive treatments are still the only option.
Advancing research in this field means working to change that.
Our approach builds on Kedrion’s expertise in plasma science, including the use of unused or under-used plasma fractions to identify new therapeutic opportunities from proteins that are not yet used in current treatments.
We are committed to expanding possibilities for patients with rare and ultra-rare diseases with the ambition to bring new possibilities to people who are still waiting for dedicated treatment options.”
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