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Swedish Orphan Biovitrum (Sobi) recently announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The phase 4 study, which is being sponsored by Sobi and Bioverativ Therapeutics, is designed to evaluate the use Eloctate™ for immune tolerance induction (ITI) therapy in hemophilia A patient with inhibitors. ITI is an effective approach to inhibitor eradication in which the body begins to tolerate a therapy after daily doses of factor are administered over time.
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TRM-201 (rofecoxib), a class of COX-2 selective non-steroidal anti-inflammatory drug (NSAID) for the treatment of hemophilic arthropathy. Rofecoxib is manufactured by Cambridge, Massachusetts-based Tremeau Pharmaceuticals. A start-up company, Tremeau is focused on developing nonopioid pain treatments for rare diseases.
The U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.
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