AstraZeneca’s rare disease drug Wainua fails Phase 3, narrowing path to $80 billion sales goal

AstraZeneca announced that its rare disease therapy Wainua did not meet its primary endpoints in a Phase 3 trial, ending plans to broaden the drug’s label for transthyretin amyloidosis cardiomyopathy.

The antisense oligonucleotide had received FDA approval in December 2023 for transthyretin amyloidosis with polyneuropathy and generated about $212 million in sales last year, far below the multi‑billion‑dollar peak revenue the company had projected.

The setback comes as AstraZeneca aims to grow total sales to $80 billion by 2030, a target that analysts had previously estimated could reach $66.8 billion. With Wainua’s growth now uncertain, the company will need positive outcomes from two other high‑risk readouts to stay on track.

Despite the disappointment, AstraZeneca’s leadership remains confident that the broader portfolio can still deliver the growth needed to reach its long‑term financial goal.