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Disease

factor X deficiency

Late-stage therapeutic development
4
Clinical trials
1
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial with recent milestones.
Clinical Milestones1View
Other updates 1
Activity timeline1

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Concizumabapproved

Approval — Alhemo is indicated for routine prophylaxis of bleeding in patients 12 years of age or mo… (2024)

Marstacimabapproved

Approval — Hympavzi is indicated for routine prophylaxis of bleeding episodes in patients 12 years o… (2024)

Etranacogene dezaparvovecapproved

Approval — Treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency)… (2023)

Valoctocogene roxaparvovecapproved

Approval — Treatment of severe haemophilia A (congenital factor VIII deficiency) in adult patients w… (2022)

Turoctocog alfa pegolapproved

Approval — Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor V… (2019)

Damoctocog alfa pegolapproved

Approval — Treatment and prophylaxis of bleeding in previously treated patients (PTPs) ≥ 7 years of… (2018)

Emicizumabapproved

Accelerated approval — Hemlibra is indicated for routine prophylaxis of bleeding episodes in patients with haemo… (2018)

Rurioctocog alfa pegolapproved

Approval — Treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia A (… (2018)

Clinical trials

2 sponsors · 1 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
4
All trials
0
Active
4
Late-stage
2
Completed
Late-stage studies

A Phase III Open, Multicentre Study to Investigate the Safety, Pharmacokinetics and Efficacy of BPL's High Purity Factor X in the Prophylaxis of Bleeding in Factor X Deficient Children Under the Age of 12 Years

Phase 3 · Completed · Bio Products Laboratory

A Phase III Open-Label Multi-Center Study of the Pharmacokinetics, Safety, and Efficacy of Coagadex in the Treatment of Adults With Acquired Factor X Deficiency Associated With Light Chain Amyloidosis

Phase 3 · Withdrawn · Kedrion S.p.A.

A Phase III Open, Multicentre Study to Investigate the Pharmacokinetics, Safety and Efficacy of BPL's High Purity Factor X in the Treatment of Severe and Moderate Factor X Deficiency.

Phase 3 · Completed · Bio Products Laboratory

A Phase III Open, Multicentre Study to Investigate the Safety and Efficacy of BPL's High Purity FACTOR X in the Treatment of Factor X Deficient Subjects Undergoing Surgery

Phase 3 · Terminated · Bio Products Laboratory

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2024emaApproval — Concizumab· Alhemo is indicated for routine prophylaxis of bleeding in patients 12 years of age or more with: •    haemophilia A (congenital factor VIII deficiency) with FVIII inhibitors.•    severe haemophilia A (congenital factor VIII deficiency, FVIII < 1%) without FVIII inhibitors.•    haemophilia B (congenital factor IX deficiency) with FIX inhibitors.•    moderate/severe haemophilia B (congenital factor IX deficiency, FIX ≤ 2%) without FIX inhibitors. source ↗
2024emaApproval — Marstacimab· Hympavzi is indicated for routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with: severe haemophilia A (congenital factor VIII deficiency, FVIII < 1%) without factor VIII inhibitors, or severe haemophilia B (congenital factor IX deficiency, FIX < 1%) without factor IX inhibitors. source ↗
2023emaApproval — Etranacogene dezaparvovec· Treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors. source ↗
2022emaApproval — Valoctocogene roxaparvovec· Treatment of severe haemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). source ↗
2019emaApproval — Turoctocog alfa pegol· Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency).Esperoct can be used for all age groups. source ↗
2018emaApproval — Damoctocog alfa pegol· Treatment and prophylaxis of bleeding in previously treated patients (PTPs) ≥ 7 years of age with haemophilia A (congenital factor VIII deficiency). source ↗
2018emaAccelerated approval — Emicizumab· Hemlibra is indicated for routine prophylaxis of bleeding episodes in patients with haemophilia A (congenital factor VIII deficiency): with factor VIII inhibitors without factor VIII inhibitors who have: severe disease (FVIII < 1%) moderate disease (FVIII ? 1% and ? 5%) with severe bleeding phenotype. Hemlibra can be used in all age groups. source ↗
2018emaApproval — Rurioctocog alfa pegol· Treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia A (congenital factor VIII deficiency). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

1 match

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

F100.83

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Coagulation factor Xvia F100.83

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Blood coagulation disorder usually inherited as an autosomal recessive trait, though it can be acquired. It is characterized by defective activity in both the intrinsic and extrinsic pathways, impaired thromboplastin time, and impaired prothrombin consumption.

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.