Familial transthyretin-related amyloidosis
Also known as Amyloidosis, hereditary, transthyretin-related, Familial TTR-related amyloidosis, Hereditary ATTR amyloidosis, transthyretin amyloidosis.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Regulatory UpdatesViewHide
- 2026-07-20CHMP positive opinion — TafamidisTreatment of hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
- 2026-07-20RegulatoryCHMP positive opinion — TafamidisTreatment of hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — For the treatment of wild-type or variant transthyretin amyloidosis in adult patients wit… (2025)
Approval — Attrogy is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (… (2025)
Approval — Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with… (2025)
Approval — Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult p… (2022)
Accelerated approval — Treatment of stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthy… (2018)
Accelerated approval — Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis… (2018)
Approval — Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients wi… (2011)
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
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
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Reference
Authoritative identity, definition & identifiers.
Amyloidosis, hereditary, transthyretin-related, Familial TTR-related amyloidosis, Hereditary ATTR amyloidosis, transthyretin amyloidosis
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Related entities are derived from literature co-mention (studied together) — associative, not causal.