Sanofi and partner Swedish Orphan Biovitrum shared positive topline results from their ongoing Phase III trial on a candidate drug for patients diagnosed with severe hemophilia A.
BioMarin Pharmaceutical shared positive results from the company’s ongoing Phase III GENEr8-1 trial on the viability of valoctocogene roxaparvovec as a treatment for patients diagnosed with severe hemophilia A.
Genentech’s Hemlibra (emicizumab-kxwh) showed no new safety signals and continued to deliver consistent bleeding control in the final analysis of a long-term, late-stage hemophilia A study.
Ahead of a planned Phase III study, Pfizer and Sangamo Therapeutics released follow-up data from its Phase I/II Alta study in hemophilia A that showed an investigational gene therapy treatment provided blood-clotting activity levels for more than one year.
San Rafael, Calif.-based BioMarin Pharmaceutical announced positive Phase III clinical trial results for the company’s gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
Bayer announced a five-year partnership with the World Federation of Hemophilia Humanitarian Aid Program, which will bring education, training and Bayer’s full portfolio of recombinant Factor VIII treatments to healthcare professionals in more than 60 countries where access to care is limited.
Bayer plans to build a Cell Culture Technology Center in Berkeley, Calif., investing $150 million in the project. The center will focus on developing biologics, an area where Bayer lags behind the company’s competitors.
Denmark-based Novo Nordisk snagged approval from the U.S. FDA for a new treatment for hemophilia A, though Esperoct will not be available in the United States until 2020.
Roche’s Hemlibra provided sustained bleed control in the largest pivotal study to date of children with a form of hemophilia, the Swiss drugmaker said.
Bayer AG’s long-acting treatment for hemophilia A won a recommendation from a European Medicines Agency (EMA) panel for the treatment of the rare genetic disorder in which blood does not clot easily.