FDA approves first drug treatment for Alexander disease
( NewsNation ) — The FDA approved the first drug to treat Alexander disease, the administration announced Thursday in a news release .
Zanvastro (zilganersen) is an injection that can be used as treatment for the disease in pediatric and adult patients.
Alexander disease can present at any point in life, with some developing the disease within the first month of infancy. It is a progressive neurological disorder that affects the brain's white matter, the network of nerve fibers in the brain that help nerve cells communicate, by damaging myelin, according to the Cleveland Clinic.
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The disease is caused by a genetic variation in the glial fibrillary acidic protein (GFAP) gene. This gene normally supports and strengthens cells in your nervous system by producing proteins that link together to form filaments. In people with Alexander disease, abnormal clumps of protein called Rosenthal fibers build up in cells damaging the myelin.
The FDA approved the drug Zanvastro, which directly targets the Rosenthal fibers that cause the disease. The drug is administered through injection into the spinal canal every three months. It works by reducing the production of the abnormal protein before it can accumulate and cause further damage, according to the news release.
The disease impacts less than one million people but can be fatal, with serious symptoms including: difficulty walking, muscle weakness, increased pressure on the brain from fluid buildup, seizures and loss of developmental milestones. Children usually develop more severe symptoms as time goes on. The most common type is infantile which presents before 2 years old, according to the Cleveland Clinic.
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"For patients with Alexander disease and their families, there have been no approved treatment options, only supportive care while the disease progresses," said Dr. Emily Freilich, director of the Division of Neurology for the FDA's Center for Drug Evaluation and Research, in the release. "Today's approval is a landmark moment for this community, offering the first therapy that addresses the underlying cause of this rare and serious disease."
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