Maria Babak, Associate Professor at City University of Hong Kong, shared an update from The Babak Lab, on LinkedIn, adding:
“A major milestone for the Sanfilippo community and another great example of AAV-based gene therapies targeting the CNS.”
Quoting The Babak Lab:
“The FDA has approved the first gene therapy for children with Sanfilippo syndrome type A.
Fayuvi (rebisufligene etisparvovec-hopf) is the first FDA-approved treatment for MPS IIIA, a rare inherited neurodevelopmental disease that progressively affects the brain and nervous system. Until now, treatment was limited to symptom management.
Key Insights
- Fayuvi is a one-time intravenous AAV9-based gene therapy.
- It delivers a functional SGSH gene, allowing cells to produce the missing enzyme sulfamidase and reduce harmful heparan sulfate accumulation.
- In children aged 2–5 years, treated patients maintained or improved cognitive function compared with an untreated historical control group.
- The therapy uses systemic AAV9 delivery to reach the central nervous system.
- Important safety considerations include thrombotic microangiopathy (TMA) and potential long-term risks associated with AAV-based gene therapy.
Conclusion
Fayuvi represents the first disease-modifying treatment for Sanfilippo syndrome type A and an important milestone for gene therapy in rare pediatric neurological diseases. Long-term safety and clinical outcomes will remain important to follow.
Image generated using Sora by OpenAI.
The FDA announcement can be found here.”
You can also read other articles about FDA Approvals on OncoDaily.