Ionis Pharmaceuticals

CHMP recommends Wainzua (eplontersen) for EU approval in hATTR-PN, citing trial benefits on TTR concentration and neuropathy. Wainua, its US counterpart, was FDA-approved in 2023, offering self-administered treatment for ATTRv-PN.

The MHRA approves eplontersen (Wainzua) for treating familial amyloid polyneuropathy (FAP) in the U.K., following U.S. approval. The therapy, developed by Ionis Pharmaceuticals and Astrazeneca, aims to slow FAP progression by suppressing the production of abnormal transthyretin (TTR) protein. Wainzua is the first self-administered FAP treatment via an autoinjector, offering monthly subcutaneous injections. The EU's CHMP recommends Wainzua's approval for FAP patients with stage 1 or 2 polyneuropathy. Clinical trial data supports Wainzua's efficacy in reducing TTR and improving quality of life, though vitamin A supplementation is advised due to potential side effects.

Ionis Pharmaceuticals and AstraZeneca's Wainzua recommended for approval by EU's CHMP for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or 2 polyneuropathy, potentially becoming the only self-administered monthly treatment in the EU for ATTRv-PN.

Wainzua (eplontersen) recommended for approval by EU's CHMP for treating hereditary transthyretin-mediated amyloidosis in adults with polyneuropathy, based on NEURO-TTRansform Phase III results showing consistent and sustained benefits in neuropathy impairment and quality of life versus placebo.

AstraZeneca's Wainzua, developed with Ionis Pharmaceuticals, recommended for EU approval to treat polyneuropathy in hereditary transthyretin-mediated amyloidosis. Phase 3 trials showed consistent benefits on serum transthyretin concentration and neuropathy impairment.

AstraZeneca's Wainzua (eplontersen) approved by MHRA for hereditary transthyretin amyloidosis (ATTRv) with polyneuropathy, reducing TTR protein production to mitigate disease progression.

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Ionis Pharmaceuticals announced FDA Fast Track designation for zilganersen, an investigational treatment for Alexander disease, with topline data expected in H2 2025. Zilganersen targets the underlying cause of the disease by inhibiting excess GFAP production. The Phase 1-3 study completed enrollment across 13 sites in 8 countries.