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CHMP Recommends WAINZUA For EU Approval To Fight Hereditary Polyneuropathy

Ionis and AstraZeneca's WAINZUA gets EU recommendation for treating hereditary ATTRv-PN via auto-injector.

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  • Oct 21, 2024

  • Simantini Singh Deo

CHMP Recommends WAINZUA For EU Approval To Fight Hereditary Polyneuropathy

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has announced that WAINZUA (eplontersen), developed in partnership with AstraZeneca, has received a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in the European Union. This recommendation is for the treatment of adult patients with hereditary transthyretin-mediated amyloidosis (ATTRv) and stage 1 or stage 2 polyneuropathy (hATTR-PN). Upon approval by the European Commission, WAINZUA would become the first and only medication available in the EU for ATTRv-PN that can be self-administered once a month using an auto-injector.


The CHMP’s recommendation is based on results from the NEURO-TTRansform Phase 3 clinical trial, which demonstrated that WAINZUA provided consistent, long-lasting benefits over 66 weeks. Patients showed significant improvement in serum transthyretin (TTR) levels, neuropathy progression (assessed via the modified Neuropathy Impairment Score +7), and quality of life measures on the Norfolk QoL-DN compared to external placebo. 


Throughout the study, WAINZUA maintained a strong safety and tolerability profile. ATTRv-PN is a progressive and often deadly disease that damages peripheral nerves, leading to motor disability within five years and, without intervention, can result in death within a decade. WAINZUA, a once-monthly RNA-targeting therapy, works by directly reducing the production of the TTR protein, which is at the core of the disease.


In December 2023, the U.S. approved eplontersen for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN), marketed under the name WAINUA™. The medication is now securing approvals in various countries around the globe, including Canada. Under a global development and commercialization partnership, AstraZeneca and Ionis are jointly promoting WAINUA in the U.S., while they pursue regulatory clearance in Europe and other regions where AstraZeneca holds exclusive rights for commercialization and development. 


Additionally, eplontersen has received Orphan Drug Designation from both the U.S. and the EU for treating transthyretin-mediated amyloidosis (ATTR). Currently, eplontersen is undergoing evaluation in the global CARDIO-TTRansform Phase 3 trial aimed at adults with ATTR cardiac amyloidosis (ATTR-CM). This trial has fully enrolled over 1,400 participants, making it the largest and most comprehensive study conducted in this patient group to date.


Brett P. Monia, Ph.D., chief executive officer, Ionis, said in a statement, "Hereditary transthyretin-mediated amyloidosis with polyneuropathy remains a progressive and debilitating disease in Europe and other parts of the world, despite currently available medicines. The CHMP recommendation is an important step toward making WAINZUA available in Europe.


He further continued, “If approved, it will be the only medicine in the EU for the treatment of transthyretin-mediated amyloidosis with polyneuropathy that can be self-administered monthly via an auto-injector. We are proud to partner with AstraZeneca whose global leadership and expertise positions our alliance to rapidly and effectively bring WAINZUA to many people living with hereditary transthyretin-mediated amyloidosis with polyneuropathy in Europe, pending the EMA's decision.”

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