Health & Bio2 min read
Health & Bio
FDA Approves Zanvastro, First Disease-Modifying Therapy for Alexander Disease
Ionis Pharmaceuticals has won FDA approval for Zanvastro, the first disease-modifying therapy for Alexander disease, a rare and deadly neurological condition, after a pivotal trial showed it kept patients' walking…
Ionis Pharmaceuticals received Food and Drug Administration (FDA) approval on Thursday for Zanvastro, the first — a treatment that alters the course of a disease rather than only easing its symptoms — for Alexander disease, a rare and deadly neurological condition. The FDA approved Zanvastro for use in both children and adults.
The approval was based on a in which walking speed, a common measure of motor function in neurologic disease, stayed stable in patients treated with Zanvastro, while patients in the control group saw a 33% decline.
The study also contained hints that treating young children with the drug can improve motor function, not just stabilize it.
Researchers found the drug was generally safe, with serious adverse events more common in the control group than among patients who received treatment.
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The story so far
- Broccoli Compound Sulforaphane Shows Early Promise Against Rare Nerve Disease
- Food and Drug Administration Approves New Pill for Rare Disease Dermatomyositis
- AstraZeneca's Heart Drug Fails to Add Benefit in Trial for ATTR-CM Patients
- Ultragenyx's Angelman Syndrome Drug Fails Late-Stage Trial
- FDA Approves Zanvastro, First Disease-Modifying Therapy for Alexander Disease
#Alexander disease#Ionis Pharmaceuticals#Zanvastro#FDA approval#rare disease#neurological disorder
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