14:I[94367,["/_next/static/immutable/chunks/4370mg0uq-myq.js","/_next/static/immutable/chunks/0q7ni_ir0mogv.js","/_next/static/immutable/chunks/0q9_4lzwjwc3f.js","/_next/static/immutable/chunks/1p8rnjckwz-06.js","/_next/static/immutable/chunks/3zikwth1q5z_-.js","/_next/static/immutable/chunks/0eigul57_x91p.js","/_next/static/immutable/chunks/3r0kxtkfnobr8.js","/_next/static/immutable/chunks/260mvqou7a4l3.js","/_next/static/immutable/chunks/14bkwvgoktej3.js"],"NewsDetailPage"] 15:T11b4,

Rare genetic disorders can cause progressive damage to vital organs and developmental functions, particularly when the underlying disease mechanism remains untreated. Gene therapy is an emerging approach that aims to address the genetic causes of certain conditions by delivering functional genetic material to patients’ cells. In paediatric medicine, such treatments may provide new options for children affected by rare and progressive diseases with limited therapeutic alternatives.

The U.S. Food and Drug Administration (FDA) has approved Fayuvi (rebisufligene etisparvovec-hopf) for paediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. The approval was announced on 17 September 2026 and represents the first FDA-approved treatment specifically designed to address the underlying course of this rare neurodevelopmental disorder. The therapy was approved for Ultragenyx Pharmaceutical, Inc.

Sanfilippo syndrome type A is an inherited condition that progressively affects the brain and nervous system. It is associated with deficiency of the enzyme sulfamidase, which is required for the breakdown of heparan sulfate within lysosomes. The resulting accumulation of heparan sulfate can contribute to progressive loss of cognitive, language and other developmental abilities. Before this approval, treatment primarily focused on managing symptoms.

Fayuvi is administered as a single intravenous infusion and uses a modified, non-infectious adeno-associated virus serotype 9 (AAV9) to deliver a functional copy of the SGSH gene into patients’ cells. This enables cells to produce sulfamidase, supporting the breakdown of heparan sulfate and reducing its harmful accumulation in the body and brain.

The safety and effectiveness of Fayuvi were evaluated in an open-label, single-arm, multicentre clinical study involving paediatric patients with MPS IIIA. The study assessed changes in cognitive scores among children aged 2 to 5 years. According to the FDA, treated patients maintained or improved cognitive function compared with an untreated historical control group.

The FDA also highlighted safety considerations associated with the treatment. Reported adverse reactions included increased liver enzymes, nausea, vomiting, fever, reduced appetite, decreased white blood cell and platelet counts, and increased amylase. Important safety warnings include the risk of thrombotic microangiopathy (TMA). The agency also noted a potential long-term risk of genetic material integrating into the genome and potentially contributing to tumour development.

Fayuvi must be administered in a healthcare setting equipped to manage infusion-related reactions. Patients are required to receive corticosteroid treatment beginning one day before infusion and continuing for at least eight weeks afterwards. The therapy received Orphan Drug, Fast Track and Breakthrough Therapy designations before approval.

The approval introduces a new treatment option for children and families affected by Sanfilippo syndrome type A while highlighting the importance of long-term monitoring and continued evaluation of gene therapy safety and outcomes.

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