Zilganersen granted U.S. FDA Fast Track designation for people …
Oct 1, 2024 · Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, …
Alexander Disease Research — End Alexander Disease
The European Medicines Agency (EMA) has granted orphan drug designation to ION373, providing regulatory and financial incentives to support the development of therapies for rare, …
Alexander’s Disease: Potential Drug Targets and Future Directions
May 31, 2025 · This review explores potential drug targets that may impact GFAP gene expression, such as STAT3, GDNF, NF-kB, LCN-2, and the LPS pathway. These drug targets …
Zilganersen Designated Breakthrough Therapy for Alexander Disease
Dec 8, 2025 · The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to zilganersen for the treatment of Alexander disease (AxD).
Positive Results Announced for Alexander Disease Clinical Trial
Sep 22, 2025 · Dear Alexander disease community, Ionis announced today positive initial results from the pivotal clinical trial (NCT: 04849741) 1 of zilganersen (ION373) in people with …
FDA Grants Breakthrough Status to Ionis’ Drug for Alexander Disease
Dec 4, 2025 · According to Brett Monia, Ph.D., chief executive officer of Ionis, these back-to-back achievements highlight the strength of the company’s innovation and the depth of its …
Zilganersen Gains FDA Fast Track for Alexander Disease, a Rare ...
Oct 9, 2024 · Recently, the FDA granted fast track designation to Ionis’ antisense oligonucleotide zilganersen as a potential treatment of Alexander disease (AxD), an ultra-rare, progressive …
Zilganersen Receives FDA Fast Track Designation for Alexander Disease
Oct 1, 2024 · The FDA's Fast Track designation is intended to accelerate the development and review of drugs that demonstrate the potential to treat serious conditions and address unmet …
Various interventions such as physiotherapy, speech therapy, nutrition, and anti-epileptic drugs can help patients maintain the best possible quality of life as the disease progresses. The …
Ionis Gets FDA Fast-Track Designation for Zilganersen in Alexander Disease
Oct 1, 2024 · The FDA's fast-track program is designed to facilitate the development and expedite the review of treatments for serious or potentially life-threatening illnesses with high unmet …