BLA for pegzilarginase in the treatment of arginase 1 deficiency (ARG1-D) accepted for priority review by the U.S. FDA
Stockholm, November 5, 2024: Immedica announces today that the Biologics License Application (BLA) for pegzilarginase in the treatment of arginase 1 deficiency (ARG1-D) has been successfully validated and accepted for priority review by the U.S. Food and Drug Administration (FDA).
Anders Edvell, CEO of Immedica commented: “We are pleased that the FDA has accepted the re-submitted BLA of pegzilarginase and granted priority review. This is a significant step towards providing a much-needed treatment option for patients with ARG1-D in the U.S. For this rare and debilitating condition there are limited therapeutic options, and we are hopeful that pegzilarginase, if approved, could offer a valuable alternative for managing the disease. We look forward to continuing to work with the FDA to potentially bring this treatment to those in need.”
About pegzilarginase
Pegzilarginase is a novel recombinant human enzyme and has been shown to rapidly and sustainably lower levels of the amino acid arginine and its toxic metabolites in plasma accompanied by improvements in clinical outcomes. Pegzilarginase is approved under the tradename Loargys® in the EU and Great Britain for the treatment of ARG1-D, also known as hyperargininemia, in adults, adolescents and children aged 2 years and older and is the first and only disease modifying treatment for ARG1-D.
About ARG1-D
ARG1-D is one of the eight urea cycle disorder (UCD) subtypes. It shares some overlapping features with other UCDs and the most prominent is the impairment in excreting nitrogen. However, in ARG1-D, hyperammonemia is generally less severe and instead these patients show spasticity, which other subtypes do not. The principal defect in ARG1-D leads to accumulation of plasma arginine and its toxic metabolites, which occurs in almost all patients with this disorder. Patients are often diagnosed in late infancy or early childhood and the symptoms include spasticity, seizures, developmental delay, intellectual disability, and early mortality.
About Immedica
Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care.
Immedica was founded in 2018 and employs today more than 140 people across Europe, the Middle East and the U.S. The main owners are the investment firms KKR and Impilo.
For more information visit www.immedica.com
Immedica contact:
Linda Holmström
Head of Communication
linda.holmstrom@immedica.com
Immedica Pharma AB
Solnavägen 3H
SE-113 63 Stockholm