News

Atomwise, a company that specializes in using artificial intelligence (AI) to develop small molecule medicines, will partner with GC Pharma to discover new ways of treating people with hemophilia. …

Mononine, a long-standing treatment to prevent and control bleeding in hemophilia B, will be discontinued due to declining demand in recent years. CSL Behring, the therapy’s manufacturer, said the decision was based on the emergence of next-generation hemophilia B treatments now in greater use. “Over time,…

Reports of translucent particles of being detected in Hemlibra (emicizumab) in 2019 do not pose a safety risk or affect the therapy’s effectiveness and, according to Roche, health authorities have agreed that the treatment’s benefit/risk profile remains unchanged. Yet, the company is proposing the use of a…

Daratumumab — a cancer therapy marketed as Darzalex — can reduce levels of factor VIII (FVIII) inhibitors in people with hemophilia type A, researchers say. Their findings were published in a letter to the editor, titled “Daratumumab rapidly reduces high‐titre factor VIII inhibitors in…

A single dose of BIVV001 is safe and effectively increases factor VIII (FVIII) activity over one month in men with severe hemophilia A, lasting longer in the body than the replacement therapy Advate, results from the EXTEN-A clinical trial suggest. Gains in half-life seen with BIVV001 “could signal…

The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…

The global pharmaceutical company Mylan has voluntarily issued a nationwide recall of four lots of tranexamic acid injection — a generic form of Cyklokapron — and of amiodarone HCl injection, according to a company announcement published by the U.S. Food and Drug Administration (FDA). The…

In rare instances, people with hemophilia can grow bones outside their skeletal tissue — with bone growth found in muscles and soft tissues — which significantly limit their range of motion, according to a recent case report. Surgery can improve the functional outcomes of patients with this abnormal bone…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Genentech has launched a program called HemeWork to support the professional development and career goals of people living with hemophilia. HemeWork was developed with input from the Hemophilia Federation of America (HFA) and bleeding disorder advocates across the U.S. to help members of the bleeding disorder community…