The National Organization for Rare Disorders (NORD) has recognized pharmaceutical company CSL Behring for pioneering Idelvion (rIX-FP), its therapeutic medicine against hemophilia B. “We are proud to present the team at CSL Behring with a 2017 Industry Innovation Award for the important work they are doing to address the needs of patients…
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Beqvez (fidanacogene elaparvovec-dzkt), a discontinued gene therapy for hemophilia B, was shown to help reduce joint bleeding in Phase 3 clinical testing. That’s according to new analysis results from an ongoing global study that assessed the treatment’s effectiveness and found positive results. Specifically, “through 15 months, 12 of…
Directly switching from Hemlibra (emicizumab) to Novo Nordisk‘s investigational Mim8 (denecimig) was well tolerated in adults and adolescents with hemophilia A, regardless of whether they had inhibitors or not. That’s according to results from the now-completed Phase 3b FRONTIER5 study (NCT05878938), which assessed the safety of switching…
The Oklahoma Medical Research Foundation (OMRF) and Shanghai RAAS Blood Products are working together on the development of new hemophilia and traumatic bleeding therapies. This is the first time OMRF has collaborated with a Chinese blood products manufacturer. In 2017, Shanghai RAAS was 25th on Forbes’ “Growth Champions”…
Xoma closed a $9.6 million deal to buy from Aptevo Therapeutics the rights to receive a payment stream for sales of Ixinity (trenonacog alfa), an approved medication used to prevent and treat bleeding episodes in people with hemophilia B. Under the terms of the deal, Xoma will…
Pfizer said it will stop development and commercialization of hemophilia B treatment Beqvez (fidanacogene elaparvovec-dzkt). “Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies…
2013 was the year that my mighty warrior Caeleb was in the hospital more than he was at home. and Christmas was a holiday that was spent in the hospital. Instead of feeling sorry for the fact that my son was in the hospital unable to walk, I…
Diabetes, which causes blood sugar levels to rise too high, is less likely to develop in young men with hemophilia than in the general male population, a U.S. study found. However, the risk of diabetes rose in older patients and in those who tested positive for hepatitis C (HCV),…
One fall evening in 1998, I looked around my dining room table in Houston and saw the faces of men and women who’d become some of my dearest friends over the past year. Laughter flooded the room as we stood in awe at what we’d accomplished. I stood, raised a…
The European Medicines Agency (EMA) will include SPK-9001 — an investigational drug for patients living with hemophilia B — in Priority Medicines (PRIME), a voluntary European regulatory initiative to support medicines that target an unmet medical need. PRIME aims to optimize development plans and speed up evaluation so that these priority medicines can…