Aeglea Bio Therapeutics Inc (NASDAQ: AGLE) has announced the submission and validation of a Marketing Authorization Application (MAA) by the European Medicines Agency (AMA) for pegzilarginase or Arginase 1 Deficiency treatment. The company’s commercialization partner in the Middle East and Europe, Immedica Pharma AB, submitted the MAA.
Pegzilarginase can regulate excess amino acid arginine concentration in ARG1-D
Pegzilarginase is a proprietary, recombinant arginase enzyme that has been demonstrated in clinical studies to regulate the excessive amino acid arginine levels in individuals with the ARG1-D. ARG1-D is a rare, progressive disorder defined by high amounts of arginine. As a result, individuals with ARG1-D have severe movement restrictions due to spasticity, developmental delay, seizures, intellectual dysfunction, and early mortality.
CEO Anthony G. Quinn said, “For progressive diseases like ARG1-D, every day that passes without an approved therapy increases the disease burden and worsens the outcome for patients and their families. If approved, we believe pegzilarginase has the potential to benefit these families. We are thrilled with the progress being made with the validation of the MAA and look forward to continuing to work with and support our partner Immedica throughout the EMA review process.”
PEACE Phase III clinical study attained 76.7% mean plasma arginine decrease
The primary objective of the PEACE Phase III clinical study was achieved with a 76.7% decrease in mean plasma arginine relative to placebo. Furthermore, 90.5% of individuals who received pegzilarginase treatment had normal plasma arginine concentrations. An improvement in the Gross Motor Function Measure Part E, a gauge of patient movement, coincided with the arginine level dropping. Clinical benefits and a persistent decrease in plasma arginine were seen in individuals with ARG1-D according to Aeglea’s Phase 1/2 and Phase 2 Open-Label Extension (OLE) findings for pegzilarginase.
Pegzilarginase has earned numerous regulatory approvals, notably Orphan Drug Designation from the European Medicines Agency, in addition to Rare Pediatric Disease, Fast Track, Orphan Drug, and Breakthrough Therapy Designations from the US Food and Drug Administration.
Additionally, Aeglea is conducting a Phase 1/2 clinical study of AGLE-177 to treat homocystinuria.