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  • Fitusiran for hemophilia receives orphan drug designation
  • by Lee, Hye-Kyung | translator Alice Kang | May 9, 2025 06:01am
Indicated for all types of hemophilia
CPAC meets unmet needs... Continuous post-marketing surveillance is required
 ¡ã Sanofi
A long-acting treatment for all types of hemophilia, administered every 2 months, has been designated as an orphan drug in South Korea.

According to the ¡°Regulations on the Designation of Orphan Drugs¡± released by the Ministry of Food and Drug Safety on the 7th, Sanofi's Fitusiran was designated as the 386th orphan drug in Korea. Fitusiran was released under the brand name Qfitlia in the U.S..

In March, the U.S. FDA approved Fitusiran as the first antithrombin inhibitor-based therapy with the convenience of a twice-monthly subcutaneous injection, for the routine prophylaxis to prevent bleeding in patients aged 12 years and older with hemophilia A or B.

Fitusiran is an siRNA (small interfering RNA) therapy
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