Ionis Pharmaceuticals, Inc. received U.S. FDA approval for its medicine for Alexander disease.
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Today, the U.S. FDA approved our medicine for Alexander disease (#AxD) marking the start of a new chapter for people and families living with this ultra-rare disease. He…
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Ionis Pharmaceuticals, Inc.
Building on our impossible firsts, we can change the course of human health.
- Industry
- Biotechnology Research
- Location
- Carlsbad, US
- Company size
- 1,001–5,000 employees
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. 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, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients.
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Latest activity from Ionis Pharmaceuticals, Inc.
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Legal & Regulatory
Ionis Pharmaceuticals, Inc. announced that the U.S. FDA has approved the first and only disease modifying treatment for Alexander disease.
Research & Knowledge
Ionis Pharmaceuticals, Inc. announced topline results from the Phase 3 Lp(a)HORIZON clinical trial in patients with cardiovascular disease and elevated levels of Lp(a) with partner Novartis.
Presence & Recognition
Ionis Pharmaceuticals, Inc. will host an investor webcast to discuss the U.S. FDA approval of its medicine for Alexander disease.
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Biotechnology Innovation Organization
Biotechnology Innovation Organization reported that Ionis Pharmaceuticals, Inc. received FDA approval for ZANVASTRO™ (zilganersen) for Alexander disease, an ultra-rare neurological disease with no prior disease-modifying treatment.
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MedChemExpress LLC
MedChemExpress LLC notes that the FDA approved Zanvastro from Ionis Pharmaceuticals, Inc. as the first FDA-approved treatment for Alexander disease and the first therapy to directly target the disease’s underlying mechanism on September 3, 2026.
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GenomeWeb announced that the antisense oligonucleotide is the first approved drug for Alexander disease
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