US FDA approves Sanofi's bleeding disorder therapy
By Sriparna Roy and Padmanabhan Ananthan
(Reuters) -The U.S. Food and Drug Administration approved French drugmaker Sanofi's hemophilia therapy, paving the way for a new type of treatment for patients with the rare blood clotting disorder, the regulator said on Friday.
US-listed shares of Paris-based Sanofi were up 1.7%.
Sanofi's subcutaneous, or under-the-skin, therapy helps prevent bleeding and lowers antithrombin, a protein that delays blood clotting, for hemophilia patients aged 12 years and older.
The therapy Qfitlia is a first-in-class for people with hemophilia A or B, with or without inhibitors, given once every two months, compared to other treatments such as Pfizer's weekly injection Hympavzi and Novo Nordisk's daily Alhemo.
"If you can take a drug every other month, that's also an incredible simplification and improvement in your quality of life," said Margaret Ragni, professor at the University of Pittsburgh.
The hemophilia market has been dominated by factor replacement therapies for decades. However, recently, gene therapies such as CSL's Hemgenix and BioMarin Pharmaceutical's Roctavian have been approved as an alternative in the United States.
People with hemophilia have a fault in a gene that regulates the production of proteins called clotting factors, causing spontaneous and severe bleeding following injuries or surgery.
Hemophilia A is caused by a lack of blood clotting factor VIII, while those with hemophilia B lack factor IX.
As many as 33,000 males are estimated to be living with hemophilia in the United States, according to the U.S. Centers for Disease Control and Prevention.
In trials, Qfitlia reduced annualized bleeding rates by 90%, compared to the control arms, showing a meaningful improvement in bleeding episodes over on-demand treatments.
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