Therapeutic Efficacy and Safety Profile of Eplontersen in Hereditary Transthyretin‐Mediated Amyloidosis: A Systematic Review

ABSTRACT Background Hereditary transthyretin‐mediated amyloidosis (hATTR) is a disorder that affects several body systems and can result in life‐threatening conditions like cardiomyopathy and polyneuropathy. For treatment of hATTR in the new medical realm, there is a breakthrough discovery of a medicine called Eplontersen, which targets the underlying genetic mutation causing the condition. This review aims to summarize Eplontersen's mechanism of action, efficacy, safety profile, and ongoing clinical trials. Methods A systematic review of the literature was conducted following PRISMA guidelines. Databases searched included PubMed, Cochrane, and Science Direct using terms related to Eplontersen and hereditary amyloidosis. Studies were selected based on defined inclusion and exclusion criteria. Results Eplontersen was approved by the FDA after the NEURO‐TTRansform phase 3 trial showed substantial safety and efficacy. The drug demonstrated a significant reduction in serum TTR levels and improvements in neurological function and quality of life in patients with hATTR. The review also explored contraindications, dosing, safety monitoring, and patient support programs. Conclusion Although data gaps remain, Eplontersen represents a promising advancement in the treatment of hATTR‐induced polyneuropathy. Future research is needed on long‐term safety, early diagnosis, personalized treatments, and combination therapies. This drug emphasizes the importance of continued research for optimal patient care and outcomes.