United States: The Qfitlia (fitusiran) medication received the US Food and Drug Administration (FDA) approval for acting as routine prophylaxis to minimize bleeding events among patients aged 12 years old and above who have either hemophilia A or B who do or do not have factor VIII and IX inhibitors.
More about the news
Qfitlia serves as a subcutaneous medication that reduces antithrombin levels, thereby increasing the blood-clotting enzyme thrombin without providing factor VIII or factor IX to patients.
Patients start treatment by receiving Qfitlia once every two months following results from the FDA-cleared INNOVANCE Antithrombin companion diagnostic test made by Siemens.
The FDA has not supported fixed dosing of Qfitlia because it leads to dangerous irregular clotting.
The FDA has accepted Liquidia Corporation's (NASDAQ: LQDA) NDA resubmission for YUTREPIA™ (treprostinil) inhalation powder to treat #PAH and #PHILD with a PDUFA goal date of May 24, 2025. See today's press release here: https://t.co/o71ooolwnW pic.twitter.com/MYT2SXAiwj
— Liquidia Corporation (@Liquidia_Corp) March 28, 2025
Thrombotic events along with gallbladder disease are listed as box warning information in the Qfitlia prescription.
The primary reported negative effects of the study included viral infection alongside nasopharyngitis and bacterial infection.
Two randomized clinical trials, including 177 adult and pediatric males with hemophilia A or B, received approval from the FDA for this testing method.
Participants who took Qfitlia antithrombin-based dosing showed a 73% decrease in the anticipated annual bleeding incidents as measured against patients getting bypassing agent on-demand treatment.
During Qfitlia treatment using antithrombin-based dosing methods for subjects without inhibitors, a 71 percent decrease in estimated annualized bleeding rate appeared compared to on-demand clotting factor concentrate treatments.
FDA Approves Qfitlia to Cut Bleeding Episodes in Patients With Hemophilia https://t.co/AFp3deX3vq
— Drugs.com (@Drugscom) April 3, 2025
What are the experts stating?
According to Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA Center for Drug Evaluation and Research, “Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,”
“Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,” Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA Center for Drug Evaluation and Research, said in a statement.
Sanofi received the approval to manufacture Qfitlia.