FDA approves experimental gene therapy directly  administered to the brain of a child

Sriansh was born with a rare genetic condition called AADC deficiency. In November 2022, at age 16 months, Sriansh became the youngest child in the world to receive a remarkably successful one-time gene therapy made by New Jersey-based PTC Therapeutics Inc. The treatment is now being marketed under the brand name KEBILIDI™.

Sriansh, now 3 years old, was part of a clinical trial that led the U.S. Food and Drug Administration to approve the treatment on Nov. 13, 2024. The new treatment represents the first gene therapy approved in the United States that is directly administered to the brain.

The therapy was developed about 15 years ago in Taiwan. Since 2010, when the first patient was treated, the therapy has been approved for commercial use by the European Union, the United Kingdom, and Israel. So far, only about 30 children worldwide have received the treatment.

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