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.

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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

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

Marstacimabapproved

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

Etranacogene dezaparvovecapproved

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

Valoctocogene roxaparvovecapproved

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

Turoctocog alfa pegolapproved

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

Damoctocog alfa pegolapproved

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

Emicizumabapproved

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

Rurioctocog alfa pegolapproved

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

Lonoctocog alfaapproved

Original application approval — Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor V… (2017)

Albutrepenonacog alfaapproved

Original application approval — Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor I… (2016)

Regulatory timeline

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

First approvals

2023emaConditional marketing authorisation — 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 ↗

2022emaConditional marketing authorisation — 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 ↗

Product approvals and authorisations

2024emaOriginal application approval — 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 ↗

2024emaOriginal application approval — 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 ↗

2019emaOriginal application approval — 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 ↗

2018emaOriginal application approval — 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 ↗

2018emaOriginal application 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 ↗

2018emaOriginal application approval — Rurioctocog alfa pegol· Treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia A (congenital factor VIII deficiency). source ↗

2017emaOriginal application approval — Lonoctocog alfa· Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency). Afstyla can be used for all age groups. source ↗

2016emaOriginal application approval — Albutrepenonacog alfa· Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX 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.