The FDA recently approved a new treatment, Qfitlia (fitusiran) from Sanofi, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies).
Qfitlia uses a different pathway in the coagulation system to rebalance hemostasis. Qfitlia is approved for those with hemophilia A and B, but, unlike most treatments for the disorder, it also can be used by patients regardless of their inhibitor status.
Traditionally, blood factors are IV administered, however, Qfitlia is administered via a subcutaneous injection making it a convenient option. Qfitlia can be administered once every two months. Dosage and frequency can also be adjusted using a companion diagnostic test.
Over the last three years, the FDA has approved six new hemophilia drugs, including three gene therapies. Qfitlia jumps into this crowded treatment landscape but it is the only therapy approved for all types of hemophilia.
The company has not announced distribution details.
The company has set a list price for Qfitlia at $642,000 annually.
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