New drug 'Wainua' for amyloid polyneuropathy receives ODD

Gained attention as the only treatment option with once-monthly administration

Demonstrated a reduction in transthyretin concentration through the Phase 3 trial

Product photo of Wainua

A new drug, 'Wainua,' for the treatment of amyloid polyneuropathy has been designated as an orphan drug in South Korea.

 

On March 5, the Ministry of Food and Drug Safety (MFDS) notified of the orphan drug The drug is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv-PN).

 

Wainua (eplontersen) is a treatment for ATTRv-PN administered through subcutaneous injection once every month at home.

 

This drug has been designed to target protein-producing liver using a new LICA technology.

 

ATTRv-PN is a progressive disease where abnormal protein deposits build up in the body's organs and tissues.

 

It is a deteriorative disease that causes neuropathy in peripheral nerves.

 

If the disease is not treated, a patient may die in 10 years.

 

Wainua was developed by AstraZeneca and Ionis Pharmaceuticals.

 

It is the only drug for the disease that can be self-administered once a month.

 

Meanwhile, the efficacy of Wainua was proven through the Phase 3 NEURO-TTRansform study.

 

The clinical results demonstrated consistent and sustained effects of Wainua at week 66 post-treatment in patients based on evaluating three primary endpoints, including transthyretin (TTR) concentration, neuropathy impairment, and quality of life (QoL).

 

Patients treated with Wainua had an 82% reduction in serum TTR concentration from the baseline, whereas the placebo group had an 11% reduction.

 

Additionally, patients treated with Wainua had a score of 0.28 points in neuropathy impairment measured by the mNIS+7(modified Neuropathy Impairment Score +7), which was an increase of 24.8 points compared to the placebo group's score of 24.8 points.

 

The results showed that Wainua stopped disease progression.

 

47% of the Wainua treatment group showed neuropathy improvement at 66 weeks, whereas 17% of the placebo group improved.