EU approves AstraZeneca and Ionis’ Wainzua for rare nerve disease

BELGIUM – AstraZeneca and Ionis Pharmaceuticals have received European Union (EU) approval for Wainzua, a drug for hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adults.

This marks the second major approval for the drug, which has been available in the United States since late 2023 under the name Wainua.

Wainzua is a once-monthly RNA-targeted medicine that works by reducing the production of transthyretin (TTR) protein in the liver, which plays a key role in ATTRv-PN.

The drug generated US $85 million in sales in 2023 and had already been approved in the United Kingdom before receiving EU clearance.

The approval was granted following a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) and is based on data from the Phase III NEURO-TTRansform trial.

Results from the trial showed that Wainzua reduced serum TTR levels by 82% over 66 weeks, compared to 11% in an external placebo group.

Additionally, 47% of patients on Wainzua showed improvements in neuropathy impairment scores, compared to 17% in the placebo group.

Brett Monia, CEO of Ionis Pharmaceuticals, emphasized the importance of this approval, stating:
“This approval offers adults with hereditary ATTRv-PN a new, self-administered treatment option that provides consistent suppression of transthyretin production and improves neuropathy impairment and quality of life.”

Competition in ATTR treatment

Wainzua enters a competitive market for ATTRv-PN treatments, where other options include:

• Amvuttra (vutrisiran) from Alnylam Pharmaceuticals, which is injected once every three months
• Vyndaqel/Vyndamax (tafamidis) from Pfizer, a daily tablet

With RNA-based treatments becoming more popular in this field, Wainzua offers an alternative option with a convenient self-injection format.

Future expansion in ATTR cardiomyopathy

While Wainzua’s current approval focuses on polyneuropathy (ATTRv-PN), AstraZeneca and Ionis are also conducting a Phase III trial (CARDIO-TTRansform) to assess its effectiveness in ATTR cardiomyopathy (ATTR-CM).

ATTR-CM affects 300,000 to 500,000 patients worldwide, compared to the 10,000 to 40,000 diagnosed with ATTRv-PN.

If successful, the trial results—expected in 2026—could expand Wainzua’s market significantly.

According to Dr. Adam Ioannou from University College London’s National Amyloidosis Centre, Wainzua could be a strong competitor in ATTR-CM if the results are as positive as the Phase III HELIOS-B trial for Amvuttra.