Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) sustainably reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. That’s according to an update on a Phase 1/2 clinical trial (NCT02576795), launched in the U.K. in 2015, that’s…
Unexpectedly high demand for the 3,000 international units (IU) vial size of Rebinyn (nonacog beta pegol) has resulted in a temporary shortage of the Novo Nordisk factor IX (FIX) replacement therapy for those with hemophilia B, according to the company. In a statement, Novo Nordisk said…
Progressive joint disease caused by repeat bleeds, a condition known as hemophilic arthropathy, affects people with moderate hemophilia in Nordic countries regardless of age, commonly causing them pain and restricting their mobility, a study found. Middle-age patients, those 41 to 50 years old, felt their overall quality of life…
The development of neutralizing antibodies, or inhibitors, against the clotting factor in replacement therapies administered to people with hemophilia may depend on the type of product used, a recent study has found. In previously untreated people with severe hemophilia A, or those who lack…
Tampa General Hospital’s cancer institute is the first healthcare center in Florida, and the third in the U.S., to successfully administer the first and only hemophilia B gene therapy, Hemgenix (etranacogene dezaparvovec), to a patient. “The infusion of this therapy is a hallmark of the TGH Cancer Institute’s…
Hemgenix (etranacogene dezaparvovec), the first and only one-time gene therapy approved for hemophilia B, has been approved in Switzerland, CSL Behring, the company marketing the therapy globally, announced. “The approval of Hemgenix in Switzerland marks an important milestone for patients, and we look forward to collaborating with…
Almost three years of treatment with Jivi (damoctocog alfa pegol) reduced the annual bleeding rate among a large, multinational group of previously treated hemophilia A patients, a real-world study reports. The number of patients without joint bleeds increased during the study and…
In the final days of 2023, an adult with hemophilia A named Maxwell became the first person in the U.S. to receive treatment with the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) — approved in June of last year — outside of clinical trials. “Receiving this treatment is liberating…
The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as a…
Health Canada has approved fidanacogene elaparvovec, formerly known as SPK-9001 and now marketed under the brand name Beqvez, for adults with moderately severe to severe hemophilia B. Developed by Pfizer, the gene therapy is under review by regulatory agencies in the U.S. and Europe. “Pfizer has…
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