Ionis gets FDA breakthrough therapy

– First and only investigational medicine for rare, often fatal neurological condition –

– On track to submit new drug application (NDA) in Q1 2026 –

 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to zilganersen for the treatment of Alexander disease (AxD), a rare, progressive and often fatal neurological condition. Over time, it can lead to loss of mobility and independence, along with difficulties walking, speaking, swallowing and breathing. There are currently no approved disease-modifying treatments for AxD. Breakthrough Therapy designation is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown preliminary clinical evidence indicating the potential for substantial improvement over available therapies.

https://finance.yahoo.com/news/ionis-receives-u-fda-breakthrough-120000903.html