Astellas's Pompe Disease Gene Therapy Trial Put On FDA Clinical Hold

  • The FDA has placed a clinical hold on Astellas Pharma Inc's ALPMY FORTIS Phase 1/2 trial following a serious adverse event (SAE) of peripheral sensory neuropathy in one of the trial participants. 
  • FORTIS trial is evaluating AT845, an investigational adeno-associated virus (AAV) gene replacement therapy in adults with Late-Onset Pompe Disease.
  • The FDA has 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 site investigator has classified the SAE as Grade 1 (mild in severity) and deemed serious due to medical significance. 
  • Astellas is working closely with the site investigator to follow the patient's clinical course and will continue gathering and reviewing all relevant data. 
  • All enrolled participants will continue to be monitored closely per the study protocol. 
  • Astellas is reviewing the potential financial impacts of this matter for the fiscal year ending March 31, 2023.
Posted In: BiotechNewsHealth CareFDAGeneralBriefsgene therapy