Thelansis

  Pfizer Inc. announced the FDA approval of HYMPAVZI™ (marstacimab-hncq) for routine prophylaxis aimed at preventing or reducing the frequency of bleeding episodes. This approval covers both adult and pediatric patients aged 12 years and older who have hemophilia A (congenital factor VIII deficiency) without factor VIII (FVIII) inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX (FIX) inhibitors. This milestone underscores Pfizer's commitment to providing innovative therapies for patients with bleeding disorders. Publish Date: 14-10-2024   Source: Pfizer Inc. Hemophilia is a severe hereditary hemorrhagic disorder, with significant types of hemophilia A (classic hemophilia or factor VIII deficiency) and hemophilia B (also known as Christmas disease or factor IX deficiency). The disorder is inherited and is characterized by a deficiency of clotting factors in the blood. Hemophilia C is a rare condition caused by a deficiency of clotting factor ...

 Hemophilia B is an uncommon congenital blood disorder characterized by a clotting factor IX (FIX) deficiency, resulting in spontaneous bleeding episodes, particularly in joints, and delayed blood clotting during external bleeding incidents. Repeated joint bleeding can lead to long-term joint deterioration, physical limitations, the necessity for joint replacements, chronic pain, and a diminished quality of life (QoL). The level of circulating FIX determines the severity of Hemophilia B (HB). The treatment approach revolves around FIX replacement therapy, which can be administered either preventively to avert bleeding episodes or reactively after a bleeding episode, known as "on-demand" treatment. Prophylactic FIX therapy is regarded as the standard of care for patients with severe and moderately severe HB. FIX is administered through intravenous injections in either standard half-life (SHL) or extended half-life (EHL) treatments every 2–3 or 7–14 days. However, the frequent ...