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# Hold Placed on Astellas Gene Therapy
- URL: https://www.fdaweb.com/hold-placed-on-astellas-gene-therapy/
- Published: 2022-06-27T12:00:00.000Z
- Updated: 2026-09-14T17:50:05.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5152176

FDA has placed a clinical hold on Astellas Pharma’s FORTIS Phase 1/2 trial following a serious adverse event (SAE) report of peripheral sensory neuropathy in one of the trial participants. FORTIS is evaluating AT845, an investigational adeno-associated virus gene replacement therapy in adults with Late-Onset Pompe Disease.

“The FDA informed Astellas that it did not have sufficient information to assess the risks to subjects and requires additional information about the recently reported SAE,” the company says. “To date, the SAE has been classified by the site investigator as Grade 1 (mild in severity) and deemed serious due to medical significance. A written explanation for the basis of the hold will be issued by the FDA and sent to Astellas within the next 30 days.”

Pompe disease is a rare, severe, autosomal recessive metabolic disease characterized by progressive muscular degeneration. The disease is caused by mutations in the alpha-glucosidase (GAA) gene that prevent the production and function of the GAA protein, the company says. “GAA is responsible for metabolizing glycogen, and dysfunction or absence of this protein results in the accumulation of glycogen in tissues, primarily in the skeletal and cardiac muscles, where it causes damage to tissue structure and function,” it says.