News

Starting or switching to prophylactic, or preventive Hemlibra (emicizumab) may lead to a significant reduction in treatment-associated expenses for hemophilia A patients in the U.S., a real-world study reported. These findings highlight the cost-benefits of using Hemlibra — and may help future cost analysis studies in assessing new…

A 70-year-old man with severe hemophilia A was treated for COVID-19 while receiving prophylactic, or preventive, treatment with Hemlibra (emicizumab) to lower his bleeding risk. This case underscores the importance of close cooperation…

As part of a Coalition for Hemophilia B contest, people with hemophilia B, their siblings, and caregivers are  invited to design a comic or coloring book that tells a story about how they manage this rare blood disease. There will be a $500 award for winners in each of…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

GenScript ProBio and Neoletix Biotechnology have entered into an agreement for clinical and commercial production of a lab-made version of coagulation factor VIII, a product that the companies hope will make the preventive treatment for hemophilia A more accessible to patients in China. Coagulation factor VIII, or FVIII,…

Optimization of investigational hemophilia B gene therapy led to more than 25 times the production of factor IX (FIX), the blood-clotting protein that is missing in people with the condition, a study has found.  According to researchers, these findings support the…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…

The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…

The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the…