News

The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer. The announcement coincides with the opening of patient enrollment for a Phase 1/2…

Forty percent of men with hemophilia who responded to a survey said the condition can weigh on their sex life, although University of California researchers cautioned that the 20-person sample was very small. Some of the respondents said they can experience bleeding from sex. In addition, the joint stiffness and joint…

Decades ago most hemophilia patients died young. Advances in treatment have led to them living about as long as the general population, which means that for the first time, doctors are facing the challenge of caring for aging hemophiliacs. A key problem is that in the days before treatment breakthroughs,…

Novo Nordisk’s recombinant coagulation factor IX nonacog beta pegol (N9-GP), under the U.S. trade name Rebinyn, has been approved by the U.S. Food and Drug Administration (FDA) to treat adults and children with hemophilia B. The company expects to launch this new treatment in the United States early in 2018. The…

Cellphones are a good way for hemophiliacs receiving care at home to relay information that doctors need to stay on top of their treatment. But a lot of patients have yet to embrace the service — and a recent online survey of 181 hemophiliacs shed some light on why. Spanish…

Chris Bombardier, the author of the “Adventures of a Hemophiliac” blog, has become the first person with hemophilia to climb Mount Everest, the world’s highest peak. That achievement on May 22 means the 31-year-old Denver native has only one mountain to go toward his goal of scaling the tallest…

The Phase 1/2 trial looking at the Factor VIII protein SHP656 as a long-acting therapeutic to treat hemophilia A did not meet a pre-defined once-weekly dosing criterion, according to Xenetic Biosciences and Shire, which are jointly developing the drug. The study demonstrated SHP656’s efficacy and pharmacokinetic data (the drug’s behavior once…

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…

The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia A developed by Sangamo Therapeutics in partnership with Pfizer. SB-525 delivers a human factor VIII cDNA construct and synthetic liver-specific promoter to the cell’s nucleus by using recombinant adeno-associated virus (rAAV). The…

Dimension Therapeutics will discontinue clinical development of DTX101, a gene therapy intended to treat moderate/severe-to-severe hemophilia B. The decision comes after a review of data from a Phase 1/2 clinical trial (NCT02618915), which assessed and the safety and dosing of DTX101. The trial…