Researchers have found that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A. The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.
Shire recently received marketing authorization from the European Commission for Adynovi [antihemophilic factor (recombinant) PEGylated], enabling access for adults and adolescents throughout Europe seeking treatment for hemophilia A. Adynovi is an extended half-life recombinant factor VIII for on-demand and prophylactic (preventive) use in patients 12 years and…
Gene therapy company uniQure released new data showing that its lead therapy AMT-060 for severe and moderately severe hemophilia B is safe and effective for up to two years, with adults requiring fewer doses of replacement therapy and showing a marked decrease in the spontaneous bleeding rate. The results support uniQure’s clinical…
Among its corporate goals for 2018 announced recently, Alnylam Pharmaceuticals said it will continue to clinically develop the investigational RNA therapy fitusiran to treat hemophilia. Fitusiran, also known as ALN-AT3SC, is an RNAi therapeutic agent that targets a protein called antithrombin — which inhibits blood clotting — while increasing production of the…
Catalyst Biosciences has amended the protocol of its ongoing Phase 1/2 clinical trial investigating the highly potent recombinant human factor IX variant CB 2679d in previously treated hemophilia B patients. The amendment has shortened the study’s duration, which is a dose-escalation study to investigate the safety and drug and organism responses (pharmacokinetics and…
Catalyst Biosciences has begun enrolling patients in a Phase 2/3 clinical trial of marzeptacog alfa (activated) as a preventive treatment for hemophilia A or B. Some patients develop immune responses — or inhibitors — to clotting factor treatments, preventing the therapies from being able to stop bleeding. Catalyst’s therapy, also known…
A web-based software to help personalize dosing regimens for some hemophilia A patients has received marketing clearance from the U.S. Food and Drug Administration (FDA). The 510(k) marketing clearance was granted to Shire, the developer, following its submission of a “premarket notification,” which is necessary if a new device is…
A novel clinical study called “The Irish Personalized Approach to the Treatment of Hemophilia (iPATH)” will seek to investigate the potential of new personalized treatment approaches in hemophilia by tailoring care based on the needs of individual patients. The collaborative study will be led by the Irish Hemophilia Society,…
Tremeau Pharmaceuticals has received guidance from the U.S. Food and Drug Administration (FDA) on how to conduct a pivotal Phase 3 trial exploring rofecoxib (TRM-201) in patients with hemophilia-associated joint pain. The advice was offered during a formal meeting with the agency, where both parties agreed on the design…
The U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, an investigational therapy for hemophilia, Alnylam Pharmaceuticals announced. “We are pleased with the FDA’s decision to lift the clinical hold, as fitusiran holds the potential to help improve the lives of people…
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