SCT800, a replacement therapy containing a man-made form of clotting factor VIII (FVIII), appears to be safe and effective at treating and preventing bleeds in adolescents and adults with severe hemophilia A, a study has found. The study, “Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted…
Effectiveness in controlling bleeds and the potential for less frequent use of a treatment are two priorities people with hemophilia place on a potential gene therapy, and both ranked slightly higher than safety, a survey study of patients in the U.S. reports. The study, “Patient preferences and…
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
In the Netherlands, children and adults with hemophilia participate in sports as much or more than people from the general population, according to a study. The findings also indicated that disease severity affected sports participation only in adults, and that children with hemophilia were more willing to participate in…
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the use of clotting factor VIII (FVIII) for up to four years in men with hemophilia A, according to the latest data from a Phase 1/2 trial and its extension study.
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
Two years of preventive treatment with Genentech’s Hemlibra (emicizumab) safely and effectively lowered the number of bleeds in people with hemophilia A and clotting factor VIII (FVIII) inhibitors, according to final data from the Phase 3b STASEY trial. Development of antibodies against the therapy (anti-drug antibodies) was…
People with severe hemophilia A who switch from standard to extended half-life replacement therapies experience fewer bleeds and require less frequent infusions, an analysis of patients in Austria indicates. Whether these gains are reflected in lower overall treatment costs, however, is less clear, its researchers added. Data from this…
The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical’s new application seeking approval of its investigational one-time gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. The application was resubmitted to EMA following a request for longer follow-up data from the Phase 3 GENEr8-1 clinical trial (NCT03370913),…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
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