The CHMP adopted a positive opinion for Wainzua (eplontersen) for the treatment of hereditary transthyretin-mediated amyloidosis and stage 1 or 2 polyneuropathy
The CHMP adopted a positive opinion, recommending the granting of a marketing authorisation 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.
Wainzua will be available as a 45 mg solution for injection in pre-filled pens. The active substance of Wainzua is eplontersen, an antisense oligonucleotide inhibitor (ATC code: N07XX21) that specifically binds to the human transthyretin (TTR) mRNA, leading to its degradation. As a result, both mutant and wild-type TTR proteins are no longer produced, which significantly reduces amyloid fibril deposits and greatly slows down the progression of ATTRv.
Wainzua has shown clinically relevant effects on both the neurological components of the disease and on quality of life. The most common side effects are vitamin A decreased and vomiting.
As part of a global development and marketing agreement, AstraZeneca and Ionis will market Wainua for the treatment of ATTRv-PN in the US and are seeking regulatory approval in Europe and other parts of the world. This agreement was recently expanded to include exclusive rights for AstraZeneca to commercialise Wainua in Latin America in addition to all other countries outside the US. Wainua was granted Orphan Drug Designation in the US and in the EU for the treatment of ATTR. Wainua will be available in the US in January 2024.
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