Sanofi: EMA approval for Pompe disease
(CercleFinance.com) - The European Medicines Agency (EMA) has agreed to examine the application for marketing authorization (MA) for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease (acid α-glucosidase deficiency).
"Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease," the group said.
Pompe disease is a rare degenerative muscle disease that can compromise a person's ability to move and breathe. It affects an estimated 50,000 people worldwide and can occur at any age, from childhood to adulthood.
The MAA is based on positive data from two clinical trials.
Copyright (c) 2020 CercleFinance.com. All rights reserved.
"Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease," the group said.
Pompe disease is a rare degenerative muscle disease that can compromise a person's ability to move and breathe. It affects an estimated 50,000 people worldwide and can occur at any age, from childhood to adulthood.
The MAA is based on positive data from two clinical trials.
Copyright (c) 2020 CercleFinance.com. All rights reserved.