Hematologic Disorders

Investigational Therapy for Hemophilia A Granted Orphan Drug Designation

The Food and Drug Administration (FDA) has granted Orphan Drug designation to SIG-001 (Sigilon Therapeutics) for the treatment of hemophilia A. SIG-001 utilizes Sigilon’s Shielded Living Therapeutics platform to implant engineered human cells to produce stable blood plasma levels of factor VIII. The cells are also shielded by Sigilon’s proprietary Afibromer biomaterials matrix that minimizes…

Tibsovo Approved to Treat Newly Diagnosed AML With IDH1 Mutation

The FDA has expanded the indication for Tibsovo to include treatment of newly diagnosed acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adult patients who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.

Esperoct Approval Provides New Treatment Option for Hemophilia A

Novo Nordisk announced that the Food and Drug Administration (FDA) has approved Esperoct (turoctocog alfa pegol, N8-GP) for the treatment of patients with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes and perioperative management of bleeding.

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