Sufferers of a very rare, progressive neurological disorder will, for the first time, have access to an FDA-approved medication, reports MassLive News.
The Food and Drug Administration has recently approved Zanvastro (zilganersen), an injection that targets the protein build-up that drives Alexander disease – a condition that, until now, had no treatment options.
Emily Freilich, director of the Division of Neurology I in the FDA’s Centre for Drug Evaluation and Research, said that patients with Alexander disease were previously offered only supportive care while the disease progressed.
She called the approval a “landmark moment” for the community, noting that bringing Zanvastro to the market marks the first time patients will have access to a therapy that addresses the disease’s root cause.
Alexander disease is classified as a rare leukodystrophy, or a group of genetic neurological disorders that damage the central nervous system. It is caused by mutations in the gene that produces glial fibrillary acidic protein, or GFAP. When the gene is altered, the protein can build up and damage the nervous system.
It affects fewer than one in a million people, but the damage it causes can be life-threatening. Common effects include seizures, difficulty walking, muscle weakness and increased pressure in the brain.
Zanvastro works by reducing elevated levels of GFAP before the build-up can cause further damage. It is administered every three months as a spinal injection by a healthcare professional.
In clinical studies, patients treated with Zanvastro showed improvements in measures of motor function, including walking ability, compared with untreated patients.
The FDA also reviewed safety and pharmacokinetic data from younger children to support its approval for patients from infancy through adulthood.
The most common side effects include vomiting, back pain, cough, headache and post-lumbar puncture syndrome. Aseptic meningitis has also been reported, and the FDA recommended patients and caregivers should discuss any concerning symptoms with their healthcare provider.
Zanvastro received several FDA designations intended to support development of treatments for serious and rare diseases, including Orphan Drug, Fast Track, Breakthrough Therapy and Rare Paediatric Disease designations.
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