Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…
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Several new treatments for hemophilia A are in development or have recently been approved. These include gene therapies like SB-525 and ASC618, which aim to induce durable clotting factor VIII activity. Other treatments include Hemlibra, a breakthrough therapy for hemophilia A without inhibitors, and Esperoct, a prophylactic treatment. Bayer is also seeking approval for BAY94-9027, a long-acting Factor VIII therapy.
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The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…
Children and adults with hemophilia A in the United States may soon be treated with N8-GP (turoctocog alfa pegol), an engineered formulation of clotting factor VIII developed by Novo Nordisk. The therapy will be available under the brand name Esperoct. The U.S. Food and Drug Administration (FDA) approved N8-GP, or…
Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…
The U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the U.S. into the safety and early efficacy of ASC618, its second-generation gene therapy for hemophilia A. A Phase 1/2 trial (NCT04676048), set to start this month, will test…
A Sangamo Therapeutics‘ gene therapy program to potentially treat hemophilia A, called SB-525, has been cleared by the U.S. Food and Drug Administration (FDA) for clinical testing in adult patients. The company announced that the FDA approved its Investigational New Drug (IND) application for SB-525, a necessary first step…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…
Geneventiv Therapeutics said it had a “positive and constructive” meeting with the U.S. Food and Drug Administration (FDA) on its plan to develop GENV-HEM, a gene therapy for hemophilia A. The meeting “represents a key regulatory milestone for Geneventiv,” Damon Race, CEO of Geneventiv, said in a…
Health Canada recently authorized Shire Pharma Canada‘s treatment Adynovate for hemophilia A patients younger than 12 based on results of a Phase 3 clinical trial. Approved Nov. 21, the injectible factor replacement therapy is available to pediatric patients for the prevention and control of bleeding episodes, as well…
The U.S. Food and Drug Administration (FDA) has restored its approval of BeneFIX as a routine prophylactic, or preventive, treatment for bleeds in children and adolescents under 16 with hemophilia B. Marketed by Pfizer, BeneFIX is a lab-made version of factor IX (FIX), the blood clotting protein that…