FDA Approves Zilganersen, the First Treatment for Alexander Disease
The US FDA has approved Zanvastro (zilganersen) injection for children and adults with Alexander disease, a rare, progressive neurological disorder caused by mutations in the GFAP gene. It is the first approved treatment for the disease.
Zilganersen is an antisense oligonucleotide that reduces production of the abnormal GFAP protein. It is given every three months by injection into the spinal canal by a trained healthcare professional. In a randomised controlled study of 49 patients aged 2 years and older, patients aged 5 and over who had walking difficulties walked significantly faster at 61 weeks than untreated patients. Younger children showed benefit on a broader assessment of motor skills.
Key points
- First FDA-approved therapy for Alexander disease.
- An antisense oligonucleotide targeting abnormal GFAP protein.
- Given by intrathecal injection every three months.
- Approved for pediatric and adult patients, based on a 49-patient randomised study and a small substudy in children under 2.
What it means for you
Alexander disease is very rare, but paediatric neurologists should know that a disease-targeted treatment now exists. Check with DRAP about whether zilganersen is available in Pakistan.
FDA Approves First Drug to Treat Alexander Disease
Summarised automatically from the official announcement. For educational purposes only — check the original source before making clinical decisions.