FDA approves first therapy for Alexander disease

The antisense oligonucleotide reduces production of the abnormal protein that accumulates in supportive cells in the brain.

To announce the FDA approval of zilganersen (Zanvastro) for treating Alexander disease.

FDA Approval: The FDA approved zilganersen injection for pediatric and adult patients with Alexander disease.
Disease Overview: Alexander disease is a rare neurologic condition caused by mutations affecting GFAP production, leading to nervous system damage.
Treatment Mechanism: Zilganersen is an antisense oligonucleotide that limits the production and accumulation of abnormal GFAP.
Clinical Study: The FDA evaluated zilganersen's efficacy and safety in a multicenter, randomized, controlled study of 49 patients aged 2 years and older.
Efficacy Results: At 61 weeks, patients aged 5 years and older showed significantly better walking speed with zilganersen compared to no treatment.
Motor Assessment: Younger patients (aged 2 to 4 years) showed improvement on a broader motor assessment, while the control group declined.
Safety Findings: The most common adverse reactions included vomiting, back pain, cough, headache, and post-lumbar puncture syndrome.

Zilganersen is the first approved therapy for Alexander disease.
The drug is administered every 3 months via spinal canal injection.
Clinical trial data supported the inclusion of patients younger than 2 years in the approved indication.

Limited clinical trial data for patients younger than 2 years due to the rarity of the disease.
Lack of a concurrent control group for younger patients.