TOKYO, June 27, 2022 - Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) announced that the US Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Ph1/2 trial following the occurrence of a serious adverse event (SAE) of peripheral sensory neuropathy in one of the trial participants. FORTIS is a clinical trial evaluating AT845, an investigational adeno-associated virus (AAV) 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. 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.
Astellas is working with the site investigator to closely follow the patient's clinical course and will continue to gather and review all relevant data. All currently enrolled participants will continue to be monitored closely per the study protocol.
“Patient safety is our top priority, and we are working closely with the FDA to determine appropriate next steps,” explained Weston Miller, M.D., Senior Medical Director, Clinical Development at Astellas Gene Therapies. “We remain committed to the safe and effective development of AT845 and will keep the scientific and patient communities informed with updates as we learn more.”
Astellas is focused on developing genetic medicines and working alongside its world-renowned partners to build a portfolio of potentially life-changing gene therapies. Astellas strives to identify, develop and deliver therapies for patients with genetic diseases who currently have few or no effective treatment options.
Astellas is reviewing potential financial impacts of this matter for the fiscal year ending March 31, 2023.
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