Ionis receives U.S. FDA Breakthrough Therapy designation for zilganersen for Alexander disease (AxD)

Core Points - Ionis Pharmaceuticals has received Breakthrough Therapy designation from the U.S. FDA for zilganersen, aimed at treating Alexander disease, a rare and often fatal neurological condition [1][2] - The company plans to submit a new drug application (NDA) in Q1 2026 [1][3] Company Overview - Ionis Pharmaceuticals is recognized for its innovative approach in developing treatments for serious diseases, including neurological conditions [3][9] - The company has a strong pipeline, including marketed medicines and investigational therapies, positioning it for revenue growth and value creation [3][9] Zilganersen Study Details - The pivotal study for zilganersen involved 54 participants aged 1.5 to 53 years across 13 sites in eight countries, primarily focusing on children due to the early onset of Alexander disease [4][5] - The study demonstrated a statistically significant improvement in gait speed, with a mean difference of 33.3% (p=0.0412) at week 61 for the 50 mg dose compared to control [2][5] Alexander Disease (AxD) Insights - Alexander disease affects approximately 1 in 1 to 3 million people globally and is characterized by progressive neurological deterioration, leading to loss of mobility and independence [7] - There are currently no approved disease-modifying treatments for Alexander disease, highlighting the significance of zilganersen's potential [7][6]