AstraZeneca Shares Dropped 1.35% Despite Wainua’s FDA Approval

The AstraZeneca plc (LON: AZN) share price dropped 1.35% despite the US FDA approving Ionis Pharmaceuticals' novel treatment, Wainua (eplontersen), designed to address the polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adult patients. 

Unique among treatments for ATTRv-PN, Wainua is the sole therapy available that patients can self-administer using an auto-injector. The endorsement from the United States Food and Drug Administration (FDA) for Wainua stemmed from the encouraging outcomes of a 35-week preliminary analysis of the NEURO-TTRansform Phase III trial. 

The data revealed that patients undergoing treatment with Wainua experienced ongoing and significant improvements in crucial areas: serum transthyretin (TTR) levels, neuropathy impairment as gauged by the modified Neuropathy Impairment Score +7 (mNIS+7), and overall quality of life as determined by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).

These promising findings, demonstrating Wainua's effectiveness over various stages of ATTRv-PN, were documented in a publication by The Journal of the American Medical Association (JAMA).

Known for causing severe peripheral nerve damage and motor disabilities within five years of onset and often leading to fatality within ten years without intervention, ATTRv-PN is a devastating condition. 

Wainua, a ligand-conjugated antisense oligonucleotide (LICA) therapy, is engineered to diminish the production of TTR protein, thereby treating both inherited and wild-type forms of transthyretin-mediated amyloidosis (ATTR).

Under a comprehensive development and commercialisation agreement, Ionis and AstraZeneca are collaborating to make Wainua available for ATTRv-PN patients in the United States. Efforts are also underway to obtain regulatory approvals across Europe and other regions.

Wainua has also been recognised as an orphan drug in the US and the European Union, signifying its importance in treating rare conditions. 

Ruud Dobber, Executive Vice-President, BioPharmaceuticals Business Unit, AstraZeneca, said: “There is an urgent medical need for new therapies for people living with hereditary transthyretin-mediated amyloid polyneuropathy. The US approval of Wainua offers a new treatment option that provides a consistent and sustained reduction in serum TTR concentration compared to baseline while halting disease progression and improving quality of life for people living with this debilitating condition.”

AstraZeneca (AZN) share price. 

The AstraZeneca share price fell 1.35% to trade at 10,366.0p from Thursday’s closing price of 10,508.0p.