Astellas Pharma (TSE:4503) has revealed that the FDA has slapped a clinical hold on its Phase 1/2 FORTIS Pompe disease study after observing a serious adverse event (SAE) — peripheral sensory neuropathy — in a trial participant.
Peripheral sensory neuropathy results from damage to nerves outside of the central nervous system. The condition can lead to pain, numbness and weakness.
The FORTIS study had focused on the experimental adeno-associated virus gene replacement therapy AT845 in adults with late-onset Pompe disease.
Pompe disease is a rare genetic disorder associated with the buildup of glycogen in cells. The disease can lead to progressive muscle weakness and reduced life expectancy. Disease progression is associated with the age of onset.
Classic infantile-onset Pompe disease occurs in the first few months of life. Non-classic infantile-onset manifests around 1 year of age while late-onset appears after that — often in adolescence or adulthood.
FDA continues to gather information to understand the potential risks to subjects in the clinical study and is also researching the recent SAE report.
The Tokyo-based company said it would work with the site investigator to monitor the patient and will continue collecting and analyzing data.
“Patient safety is our top priority, and we are working closely with the FDA to determine appropriate next steps,” said Dr. Weston Miller, senior medical director, clinical development at Astellas Gene Therapies, in a statement. “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 shares were up 1.57% to ¥2,197.00.
Filed Under: Cardiovascular, Cell & gene therapy, Neurological Disease