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Factor VIII (ReFacto AF / Xyntha)

✓ Approved

Sobi · F8 · Recombinant Proteins

What is Factor VIII?

Factor VIII is a recombinant proteins developed by Sobi. It is approved for therapeutic indications via injectable (others) or intravenous (iv).

Drug Profile

Brand NamesReFacto AF, Xyntha
CompanySobi
Drug ClassRecombinant Proteins, Cell-based Therapies
Molecular TargetF8
RouteInjectable (Others), Intravenous (IV)
StatusApproved
Sources: ClinicalTrials.gov, Sobi

Mechanism of Action

Molecular Targets

Factor VIII acts on 1 molecular target:

F8coagulation factor VIII (AHF, FVIII)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

Factor VIII is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersFactor VIII deficiency✓ Approved

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Late Wilate Prophylaxis Initiation in Adult Twins with Type 3 von Willebrand Disease: Overcoming Historical Barriers and Improving Clinical Outcomes.

Sierra Cristina C, Ojinaga Maria José MJ, Insunza Lucia L, Pinedo Gorka G et al.

Prophylaxis with a plasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII) 1:1 concentrate (wilate) is effective in the treatment of severe type 3 von Willebrand disease (VWD). However, uptake of prophylaxis with factor concentrates remains limited due in part to the treatment burden and historical concerns over blood product safety. We describe clinical cases of two adult twin brothers with severe type 3 VWD and a history of hepatitis C acquired from childhood plasma-derived products, who initiated once-weekly pdVWF/FVIII 1:1 concentrate prophylaxis after years of on-demand therapy. Both patients completed 12 months of prophylaxis, which led to a marked improvement in bleeding control and daily functioning, and a decrease in treatment burden compared with on-demand treatment. Their experiences highlight the significant enhancement in quality of life achieved through the adoption of regular prophylaxis. Overcoming long-standing concerns about product safety allowed them to embrace preventive care and realize its benefits in managing their severe VWD.

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Parkinson's Disease (PD) is a neurodegenerative disorder marked by the progressive loss of dopaminergic neurons within the dopamine nigrostriatal pathway. Current pharmacological treatments provide only symptomatic relief and are associated with several adverse effects. While indirect evidence strongly supports the therapeutic potential of metabotropic glutamate receptor 3 (mGlu3) in PD, pharmacological limitations have hindered progress in the field. In this study, we present the first functional characterization of a tetrahydroisoquinoline derivative acting as a dual agonist/positive allosteric modulator (Ago/PAM) of mGlu2/3. This compound exhibits slightly higher potency for mGlu3 compared to mGlu2, along with notable neurotrophic properties. The study employed complementary in vitro and in vivo approaches, using a cellular model of PD and healthy male C57BL/6 mice. The novel Ago/PAM shows equal affinity for mGlu2 and mGlu3 and greater efficacy at mGlu3 receptors in recombinant cells. Exposure to the mGlu2/3 Ago/PAM protects the human neuroblastoma cell line SH-SY5Y from 6-hydroxydopamine (6-OHDA)-induced cell death by modulating neurotrophic signaling pathways, including the Mitogen-Activated Protein Kinase/Extracellular Signal-Regulated Kinase (MAPK/ERK) and Phosphatidylinositol 3-Kinase (PI3K)-AKT pathways. Moreover, this treatment increases the expression of Glial Cell Line-Derived Neurotrophic Factor (GDNF), a key factor for dopaminergic neuron survival. Interestingly, pre-treatment with ML 337, a negative allosteric modulator for mGlu3, abolishes mGlu2/3 Ago/PAM-induced neuroprotective effects. In vivo administration of mGlu2/3 Ago/PAM upregulates GDNF and Brain-Derived Neurotrophic Factor (BDNF) expression, while activating MAPK/ERK and PI3K-AKT pathways in mouse brain regions involved in PD pathophysiology, such as the striatum, the hippocampus, and the prefrontal cortex. Collectively, these findings identify this tetrahydroisoquinoline-derived compound as a novel mGlu2/3 Ago/PAM with preferential activity at mGlu3 and provide its first functional characterization, demonstrating robust neurotrophic and neuroprotective effects. By combining agonist and positive allosteric modulation with mGlu3 selectivity, this compound represents a translationally promising strategy to counteract neurodegeneration and restore neuronal-glial homeostasis in PD. These results, though preliminary, support the therapeutic potential of this compound and, more broadly, of mGlu3-targeted approaches, laying the foundation for future in vivo studies to validate efficacy, safety, and receptor specificity.

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Kang Hoseong H, Cho Sungmin S, Yu Su-Jeong SJ, Lee Gil-Yong GY et al.

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Association between elevated C-reactive protein and mortality, healthcare resource utilization and costs in patients with atherosclerotic cardiovascular disease and/or chronic kidney disease.

Ray Kausik K KK, Barkas Fotios F, Huisman Eline L EL, Mathisen Jimmi J et al.

Atherosclerotic cardiovascular disease (ASCVD) and chronic kidney disease (CKD) impact health and healthcare systems. Inflammation is a potential modifiable risk factor for both conditions. The case for evaluating the modification of inflammation in these conditions could be strengthened by evidence that systemic inflammation [C-reactive protein ≥2 mg/L] was associated with a considerable health burden. A retrospective observational cohort study of 179 261 individuals in mutually exclusive cohorts (ASCVD only, CKD stages 3-4 only, ASCVD + CKD stages 3-4) and a reference cohort (no ASCVD or CKD) with ≥1 C-reactive protein measurement after diagnosis. Outcomes included all-cause mortality, healthcare resource utilization (HCRU), and direct healthcare costs. Chronic kidney disease stages 3-4, ASCVD only, and ASCVD + CKD stages 3-4 cohorts compared with the reference cohort were associated with increasing all-cause mortality (death/100 000 person-years) of 1637, 2090, 5163, and 946, respectively. HCRU events per person per year (PPPY) and mean direct costs PPPY were 20.4, 21.4, 34.2, and 8.2 and £2593, £3098, £6810, and £1144, respectively. Within cohorts, all-cause mortality increased progressively across C-reactive protein quartiles (Q) and was highest in Q4 among the ASCVD + CKD stages 3-4 cohort, with a hazard ratio (Q4 vs. Q1) of 2.75 in this cohort compared with 1.22 in the reference cohort. While the clinical impact of targeting inflammation is being evaluated in ongoing trials, this real-world study demonstrates that assessing inflammation in patients with ASCVD and CKD stages 3-4 can help to identify a population with a high healthcare burden.

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