New Drug: Vutrisiran for transthyretin amyloidosis

Vutrisiran for transthyretin amyloidosisActive ingredient: vutrisiran Brand name (sponsor): Amvuttra (Medison Pharma) Presentation: clear, colourless-to-yellow sterile solution in single-use prefilled syringe with needle containing vutrisiran sodium equivalent to 25 mg vutrisiran in 0.5 mL solution Route of administration: subcutaneous injectionApproved indication: treatment of hereditary transthyretin amyloidosis (hATTR) in adult patients with stage 1 or stage 2 polyneuropathy Background: Hereditary transthyretin amyloidosis (hATTR, also known as ATTRv) is a rare, autosomal dominant, multisystem condition caused by mutations or variants in the transthyretin (TTR) gene.TTR is a protein predominantly produced in the liver that transports thyroxine and retinol-binding proteins, so affects serum vitamin A concentration.Genetic variants in the TTR gene result in misfolding of the TTR protein, destabilising the structure, and ultimately resulting in the formation of abnormal, insoluble amyloid fibrils.These amyloid fibrils deposit throughout the body, predominantly in extracellular tissue, leading to multisystem disease, debilitating morbidity and high mortality.1,2 Whilst the peripheral nervous system and heart are most commonly affected, organ involvement may include the kidney, eyes, central nervous and gastrointestinal systems; hence, clinical presentation is widely variable.1,2 Adverse effects: Although most patients (97.5%) in the vutrisiran group reported adverse events, the