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Ionis receives U.S. FDA Breakthrough Therapy designation for zilganersen for Alexander disease (AxD)
– First and only investigational medicine for this rare, often fatal neurological condition – – On track to submit new drug application (NDA) in Q1 2026 –
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"\n– First and only investigational medicine for this rare, often fatal neurological condition –\n\n– On track to submit new drug application (NDA) in Q1 2026 –\n\n CARLSBAD, Calif.--(BUSINESS WIRE)--\nIonis 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.\n\n“People living with Alexander disease have gone far too long without a treatment capable of changing the course of their disease, which makes this Breakthrough Therapy designation particularly meaningful,” said Holly Kordasiewicz, Ph.D., senior vice president of neurology, Ionis. “Our pivotal zilganersen study provides the first evidence that an investigational treatment can modify the underlying disease and improve outcomes, representing an important step forward for the Alexander disease community. We are deeply grateful to the individuals, families, caregivers and investigators who made this progress possible, and we are committed to working closely with the FDA to bring this potential treatment to those in urgent need.”\n\nThe FDA’s designation is supported by the topline results from the pivotal study of zilganersen in children and adults living with AxD. In the study, zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test (10MWT) compared to control at week 61 (mean difference 33.3%, p=0.0412) with favorable safety and tolerability. Zilganersen also demonstrated consistent benefit in key secondary endpoints.\n\n“Receiving back-to-back Breakthrough Therapy designations for olezarsen in severe hypertriglyceridemia and zilganersen in Alexander disease demonstrates how Ionis’ innovation can make...