Immedica Pharma announced that the FDA has granted accelerated approval of Loargys® (pegzilarginase-nbln), an arginine specific enzyme indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction.
This indication is approved under accelerated approval based on reduction of plasma arginine. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
"Today's FDA accelerated approval of LOARGYS is an important milestone for Immedica and for patients and families affected by Arginase 1 Deficiency in the U.S.," said Anders Edvell, CEO of Immedica. "This outcome is the result of collaborative efforts across the entire ARG1-D community including patients, advocacy groups, researchers, and clinicians. We are proud to be able to deliver a treatment option for patients and families who have long awaited progress."
ARG1-D affects an estimated 250 people living in the U.S. and current standard of care relies primarily on symptom management, including dietary protein restriction, arginine-free amino acid supplementation and nitrogen scavenging agents if necessary.
"Until now, the care of patients with Arginase 1 Deficiency has been limited to symptomatic management and strict dietary control. The accelerated approval of LOARGYS offers a fundamentally new approach that addresses the enzyme deficiency itself. Since persistently elevated arginine and its metabolites have been reported to be the proximal or direct driver of disease progression, this is a major advancement in metabolic medicine" said Dr. Stephen Cederbaum, Professor Emeritus of Human and Medical Genetics at UCLA.
LOARGYS is the first and only treatment to address persistently elevated levels of plasma arginine, the primary driver of ARG1-D.
Christine Zahn, founder and director of Arginase 1 Deficiency Foundation added, "Today marks a pivotal moment for the ARG1-D community and the physicians who support us. While the journey to this day was neither short nor simple, LOARGYS offers real hope for a better future and exemplifies what is possible when progress driven by empathy, collaboration, and relentless advocacy to address patients in need."
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