News

New preclinical data on one of Catalyst Biosciences’ lead candidate therapies — marzeptacog alfa activated (MarzAA), an engineered clotting Factor VIIa (FVIIa) given as an under-the-skin injection — support its potential to treat acute bleeds in people with hemophilia and its ability to be used safely in combination with Hemlibra…

Enzyre is teaming up with Takeda to develop a diagnostic device that will allow people with hemophilia to determine their blood coagulation status at home. Under the collaboration agreement, the Dutch diagnostics company Enzyre will receive funding from Takeda to optimize its Enzypad technology. The goal is…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…

As part of a new partnership focused on hemophilia treatment and management, members of insurer Highmark will receive medication exclusively from Option Care Health, Soleo Health, and the Hemophilia Center of Western Pennsylvania. The collaboration that begins in January is aimed at keeping down costs associated with…

A programmable mini-pump able to provide regular doses after surgery of FVIIa to hemophilia A patients with inhibitors — those resistant to standard FVIII replacement therapy — successfully maintained expected levels of blood clotting factors, a small study reports. Attending surgical nurses also voiced satisfaction with the pump’s use, reporting a…

A lower dose of regular, preventive therapy with factor VIII concentrates was better at reducing bleeding events and improving joint function than on-demand treatment in children with hemophilia in Thailand, a study shows. The study, “Prophylactic vs episodic treatment to prevent bleeds and preserve joint function…

BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A. The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly…

Pregnant women who are hemophilia B carriers have more than three times the risk of bleeding following childbirth than non-carriers, a register-based study done in Sweden has found. In contrast, researchers found no differences in the risk of postpartum bleeding between hemophilia A carriers and non-carriers, or in the…

Citing Soleo Health’s clinical expertise and data-reporting capabilities, U.S. national insurer Highmark has tapped the specialty pharmacy and infusion services provider to help care for its members who have hemophilia. An independent licensee of the Blue Cross Blue Shield Association, Highmark covers about 190 hemophilia patients.