The European Medicines Agency (EMA) has validated Aeglea BioTherapeutics Inc's (NASDAQ: AGLE) marketing application seeking approval for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D).
Arginase deficiency is an inherited disorder that causes the amino acid arginine (a building block of proteins) and ammonia to accumulate gradually in the blood.
Ammonia, formed when proteins are broken down in the body, is toxic if levels become too high.
Patients experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability, and early mortality.
The application was submitted by Immedica Pharma AB, Aeglea's commercialization partner in Europe and the Middle East.
The EMA has granted pegzilarginase orphan drug designation.
Immedica's submission includes data from multiple clinical studies, including the PEACE Phase 3 study and its ongoing long-term extension, a Phase 1/2 trial, and an open-label extension study.
Results showed that pegzilarginase could rapidly and sustainably lower arginine levels and improve mobility.
In the PEACE study, most treatment-emergent adverse events were mild or moderate, and there were no discontinuations due to treatment-emergent adverse events.
Price Action: AGLE shares are up 72.71% at $0.87 during the premarket session on the last check Friday.
See more from Benzinga
Don't miss real-time alerts on your stocks - join Benzinga Pro for free! Try the tool that will help you invest smarter, faster, and better.
© 2022 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.