New Phase 3 trial results of CSL Behring’s hemophilia B therapy candidate Idelvion show promise in terms of reduction of bleeds, prolonged usage, and effectiveness at low doses. The poster detailing the data, “Long-term safety and efficacy of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients…
Key findings from clinical trials of three recombinant coagulation factors as new hemophilia treatments, presented at last year’s Congress of the International Society on Thrombosis and Haemostasis (ISTH 2015), have been published in a supplemental issue of the journal Thrombosis Research. The results were presented in a session…
The US Food and Drug Administration has granted Breakthrough Therapy Designation to biotech Spark Therapeutics and pharmacology giant Pfizer for the hemophilia B treatment SPK-9001. The designation allows expedited development and FDA review of the gene therapy currently undergoing a Phase 1/2 trial as a potential one-time therapy. The trial is recruiting patients.
Biogen and Sobi announced that they will be presenting updated and long-term data on two approved hemophilia treatments, Eloctate (marketed as Elocta in Europe) for hemophilia A, and Alprolix for hemophilia B, at the World Federation of Hemophilia (WFH)’s 2016 World Congress next week. Specifically, data from separate Phase 3 extension studies evaluating each treatment’s safety and…
CSL Behring announced that new data from studies of two approved treatments for hemophilia — Idelvion and Afstyla, both recombinant coagulation factors — will be presented at the XXXII International Congress of the World Federation of Hemophilia (WFH) 2016 next week. CSL will present six posters highlighting Idelvion, a long-acting recombinant albumin fusion…
Researchers reviewed the current hemophilia research that focuses on the development of improved longer-acting factor replacement therapies, particularly PEGylated products. The new clinical research advances lead the scientific community to look at such developments with optimism, but also some caution. The study, “Potential role of a new PEGylated recombinant…
The European Medicines Agency (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has initiated a review of factor VIII-containing medicines, to evaluate the risk of inhibitor protein development in patients starting treatment for hemophilia A, according to a press release. This review, which will cover all medicines containing factor VIII authorized in the…
A recent study involving adolescent and young adult patients with hemophilia, to evaluate racial and ethnic disparities in following recommended clotting factor treatment regimens, chronic pain and quality of life, indicated that non-white patients report more chronic pain and worse quality of life. The research paper, “Racial Differences in Chronic…
A new study from Finland demonstrated that neutralizing antibodies (inhibitors) can develop in people with untreated hemophilia, and that early intensive prevention might prevent bleeding and inhibitor development. The research report, titled “Inhibitor development in previously untreated patients with severe haemophilia A: a nationwide multicentre study in Finland,” was published in…
Joint problems caused by chronic bleeding often trouble people with hemophilia, especially in the knees, ankles, and elbows. A paper by scientists in Spain and the U.S. reviewed existing literature on knee treatments for people with this disease, focusing on best current practices — including a preventive, if inconveniently lifelong, therapy — and future…
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