Disease

Congenital factor II deficiency

Also known as Dysprothrombinemia, Hypoprothrombinemia, Prothrombin deficiency.

Associated genes

Related proteins

What's happening now

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

No activity recorded in this window. Try a wider timeframe.

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)

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.

F20.80

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.

Prothrombinvia F20.80

Reference

Authoritative identity, definition & identifiers.

Synonyms

Dysprothrombinemia, Hypoprothrombinemia, Prothrombin deficiency

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Related entities are derived from literature co-mention (studied together) — associative, not causal.