HEM News Briefs

subcutaneous injection

Rondoraptivon Pegol Increases Factor VIII Half-Life and Levels in Hemophilia A Patients

A new study has found that the von Willebrand factor (VWF)-binding aptamer rondoraptivon pegol increased the half-life of substituted factor VIII (FVIII) as well as endogenous FVIII levels in patients with hemophilia A. The study, published in Blood, also found that the patients tolerated the subcutaneous treatment well. “Recently, rondoraptivon pegol (previously BT200), a VWF-binding…

Young Children Drawing

New Approach Could Improve Mental Health Screening in Hemophilia

A team from St. Jude Children’s Research Hospital in New York and Vanderbilt University Medical Center in Nashville, Tennessee, have developed an approach to help identify children with hemophilia at risk for attention-deficit/hyperactivity disorder (ADHD). “A reliable method of screening that leads to early identification of [inattention (IN)]/[hyperactivity/impulsivity (HI)] can facilitate early intervention in the…

clinical research

Cancer Diagnosis in Hemophilia Trial Likely Unrelated to Experimental Therapy

BioMarin will proceed with phase 3 study of the investigational gene therapy BMN 270 (valoctocogene roxaparvovec) for the treatment of adults with severe hemophilia A despite a participant being diagnosed with B-cell acute lymphoblastic leukemia (B-ALL). The diagnosis was confirmed approximately 3 years after the participant received the dose of BMN 270. Early assessment of…

clinical trial

Phase 3 AFFINE Study of Novel Gene Therapy for Hemophilia Reopens Recruitment

The Pfizer and Sangamo Therapeutics phase 3 AFFINE study assessing the efficacy and safety of giroctocogene fitelparvovec, a novel gene therapy for patients with moderately severe to severe hemophilia A, has reopened recruitment. The reopening, announced by news release, will resume enrollment this month and dosing is expected to restart in October. Giroctocogene fitelparvovec is…

subcutaneous injection

FDA Grants Orphan Drug Designation to Centessa’s SerpinPC for Hemophilia B

The US Food and Drug Administration (FDA) has granted Orphan Drug designation to Centessa Pharmaceutical’s SerpinPC , a new activated protein C (APC) inhibitor designed to treat hemophilia B. The designation, announced by news release, follows the results of a phase 2a study showing that SerpinPC at the highest dose reduced the median annualized bleeding…

coronavirus

The Risk of Extracorporeal Circuit Clotting in Patients With COVID-19 on Hemodialysis

An increased risk of extracorporeal circuit clotting (ECC) has been reported in patients with coronavirus disease 2019 (COVID-19) admitted to the intensive care unit who require continuous renal replacement therapy. Recognizing that ECC is a known issue among patients with chronic kidney disease undergoing hemodialysis (HD), which is thought to be due to turbulent blood…

european commission

EU Grants Conditional Authorization to Valoctocogene Roxaparvovec for Hemophilia

The European Commission has granted conditional marketing authorization to valoctocogene roxaparvovec (Roctavian™), a novel gene therapy for patients with severe hemophilia A, BioMarin Pharmaceutical Inc. announced. “This approval in the [European Union] represents a medical breakthrough in the treatment of patients with severe hemophilia A that expands the conversation between a patient and physician on…

joint degeneration

Biomarkers Could Identify Patients at Risk of Developing Hemophilic Arthropathy

The plasma concentration of citrullinated histone, a marker of neutrophil extracellular traps, is higher in patients with hemophilia than in people without a congenital bleeding disorder, a new study published in the Journal of Clinical Medicine found. Moreover, high serum levels of osteocalcin, a bone tissue formation marker, are more common in patients with hemophilia…

FDA-Priority-Review

FDA Grants Priority Review to Efanesoctocog Alfa for the Treatment of Hemophilia A

Sanofi Global Health announced that the US Food and Drug Administration (FDA) has granted priority review to the Biologics License Application (BLA) for efanesoctocog alfa, a novel investigational recombinant factor VIII replacement therapy for people with hemophilia A. The review, announced by news release, has an FDA target decision date of February 28, 2023. “Factor…

blood cells

Activated Prothrombin Complex Concentrate as a Hemostatic Agent in Hemophilia A

Activated prothrombin complex concentrate (aPCC) appears to be safe and highly effective as a hemostatic agent for acute bleeds due to acquired hemophilia A, according to a recently published study in the European Journal of Hematology.  Acquired hemophilia A is a rare autoimmune disease that mainly affects patients over the age of 60 years. It is…

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