- Source: Eplontersen
Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. It is a transthyretin-directed antisense oligonucleotide. It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca.
Eplontersen was approved for medical use in the United States in December 2023 and in the UK in October 2024.
Medical uses
Eplontersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Side effects
The most common adverse reactions include decreased vitamin A and vomiting.
Society and culture
= Legal status
=In October 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Wainzua, intended for the treatment of adults with hereditary transthyretin-mediated amyloidosis (ATTRv) and stage 1 or 2 polyneuropathy. The applicant for this medicinal product is AstraZeneca AB.
= Names
=Eplontersen is the international nonproprietary name.
References
External links
Clinical trial number NCT04136184 for "NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy" at ClinicalTrials.gov
Clinical trial number NCT01737398 for "Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy" at ClinicalTrials.gov
