Credit: Getty Images. The Agency stated that it “erred in approving the indication for routine prophylaxis in children with hemophilia B” due to the Orphan Drug exclusivity. The Food and Drug ...

Expanded indication includes patients under 12 years of age, based on Phase 3/4 data demonstrating safety and efficacy in previously treated patients in this age group "We are pleased to secure ...

Hemophilia A and B involve deficiencies in coagulation factors VIII and IX, with traditional treatments requiring frequent infusions. Nonfactor therapies, including Hemlibra and rebalancing agents, ...

Hemophilia B causes a deficiency in factor IX, a protein necessary for blood clotting. Replacement factor IX therapy is the gold standard treatment, but novel and genetic therapies are showing promise ...

Centessa Pharma's SerpinPC, a subcutaneous biologic inhibitor, shows promising results in reducing bleeding rates in hemophilia B, with a favorable safety profile. Despite the stock doubling in value, ...

A single infusion of verbrinacogene setparvovec resulted in normal factor IX levels among all 10 men who received the investigational gene therapy for moderate to severe hemophilia B, study results ...

The indications, pharmacokinetics, and therapeutic guidelines for available coagulation products are reviewed. Patients with hemophilia, von Wille-brand's disease (VWD), or acquired inhibitors to ...

Please provide your email address to receive an email when new articles are posted on . The FDA approved etranacogene dezaparvovec-drlb, a single-dose gene therapy for treatment of adults with ...

Hemophilia B is an X-linked recessive hemorrhagic disorder caused by a deficiency or abnormality of clotting factor IX. Human factor IX is a vitamin-K-dependent multidomain glycoprotein composed of ...