Published 5 days ago • loading... • Updated 1 day ago

FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors

The U.S. Food and Drug Administration approved Qfitlia 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.
Alnylam scientists discovered Qfitlia, which lowers antithrombin to promote thrombin generation, and Sanofi obtained global development and commercialization rights to Qfitlia under a license and collaboration agreement.
Clinical trials with 177 patients showed that Qfitlia, administered subcutaneously starting once every two months, resulted in a 73% reduction in estimated annualized bleeding rate in participants with inhibitors and a 71% reduction in those without inhibitors compared to on-demand treatments.
According to Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA's Center for Drug Evaluation and Research, the approval of Qfitlia is significant because it can be administered less frequently than other existing options and highlights continued efforts to improve the lives of patients with hemophilia.
Qfitlia carries boxed warnings for thrombotic events and gallbladder disease and a warning about liver toxicity, necessitating liver blood test monitoring; some patients may require gallbladder removal, and the most common side effect is viral infection.

Insights by Ground AI

Does this summary seem wrong?

32 Articles

All

Left

Center

Right

Bloomberg

Lean Left

Sanofi’s Easier to Use Hemophilia Drug Approved by US FDA

Sanofi’s treatment for hemophilia that can be administered as infrequently as once every other month, was approved by the US Food and Drug Administration, offering patients greater choice as the market for therapies grows.

5 days ago·United States

Read Full Article

The Berkshire Eagle

+22 Reposted by 22 other sources

Center

FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors

Medication Can be Given Up to Once Every 2 Months

5 days ago

Read Full Article

European Medical Journal

Entering a New Era in Protection in Haemophilia A

Haemophilia A (HA), defined by factor VIII (FVIII) levels ≤40 IU/dL, is a chronic condition with consequences beyond bleeding...

1 day ago

Read Full Article

GEN - Genetic Engineering and Biotechnology News…

RNAi Therapeutic for Hemophilia A or B Gets FDA Approval

Late last week, the FDA approved Qfitlia (fitusiran) 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). “Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,”…

2 days ago

Read Full Article

MyChesCo

FDA Approves Qfitlia: A Breakthrough Treatment Revolutionizing Hemophilia Care

WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has announced the approval of Qfitlia (fitusiran), a treatment designed to prevent or reduce the frequency of bleeding episodes in individuals with hemophilia A or hemophilia B. This approval applies to both adults and pediatric patients aged 12 years and older, including those with or without factor VIII or IX inhibitors. “Today’s approval of Qfitlia is significant for patients with…

2 days ago

Read Full Article

Scienmag: Latest Science and Health News

Comparative Efficacy of Prothrombin Complex Concentrate Versus Frozen

In a groundbreaking development that has significant implications for cardiac surgery practices, a recent unblinded randomized clinical trial has found that prothrombin complex concentrate (PCC) exhibits superior hemostatic efficacy compared to frozen plasma. This study addresses a critical need for effective coagulation factor replacement among patients experiencing bleeding during cardiac procedures. The findings from this trial could alter th…

4 days ago

Read Full Article

Think freely.Subscribe and get full access to Ground NewsSubscriptions start at $9.99/year