Familial Amyloidosis

• EMA approval · 2026-07-07
  EMA Authorised: Onpattro (patisiran sodium) — Alnylam Netherlands B.V.

  EMA has authorized Onpattro (patisiran sodium) for familial amyloidosis, with orphan designation and accelerated assessment.

• CLINICAL_TRIALS trial_result · 2026-03-24
  Clinical Trial Update: Comprehensive Program for Hereditary Transthyretin Amyloidosis

  Clinical trial update announcing a comprehensive program for hereditary transthyretin amyloidosis that is currently recruiting.

• CLINICAL_TRIALS research · 2025-06-04
  Clinical Trial Update: Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

  A clinical trial is recruiting to study subclinical transthyretin cardiac amyloidosis in individuals carrying the V122I TTR mutation.

• EMA approval · 2025-03-25
  EMA Authorised: Onpattro (patisiran sodium) — Alnylam Netherlands B.V.

  The European Medicines Agency authorised Onpattro (patisiran) for familial hereditary transthyretin-mediated amyloidosis with orphan designa…

• CLINICAL_TRIALS trial_result · 2021-12-21
  Clinical Trial Update: Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant [Phase 3]

  Phase 3 trial evaluating patisiran in hereditary transthyretin-mediated amyloidosis patients with disease progression after liver transplant…

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• CLINICAL_TRIALS trial_result · 2017-03-17
  Clinical Trial Update: The Effect of Diflunisal on Familial Amyloidosis [Phase 2, Phase 3]

  This update reports the completion and results posting of phase 2 and phase 3 trials evaluating diflunisal for familial amyloid polyneuropat…