Arginase 1 Deficiency News
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 [...]
Immedica initiates a phase 3 pediatric study for Loargys® (pegzilarginase) in arginase 1 deficiency
Loargys is approved in the EU and Great Britain for the treatment of ARG1-D in adults, adolescents and children aged 2 years and older. The initiation of a clinical study in children below 2 years of age is of high importance in this progressive disease exposing patients to elevated toxic levels of arginine from birth.
Immedica presents new data highlighting treatment benefits of Loargys® (pegzilarginase) in arginase 1 deficiency at the SSIEM congress
Stockholm, September 3, 2024: Immedica announces today that new scientific data on Loargys® (pegzilarginase), the first disease modifying treatment in arginase 1 deficiency, has been accepted at the 2024 Annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Porto, Portugal on 3-6 September.
Massachusetts man with rare disease denied experimental drug treatment
"It's been a long one," Alexandra Eaton told WBZ-TV from her Marshfield home. "It's been challenging, but he's persevered." You can see the love in Eaton's eyes, looking at her son, whose care and comfort consumes her entire life. Josh, who is 26, lives with rare and debilitating Arginase 1 Deficiency.
Loargys® (pegzilarginase) approved in the EU for treatment of arginase 1 deficiency (ARG1-D)
Stockholm, December 18, 2023: Immedica today announces that the European Commission has granted marketing authorization of Loargys® (pegzilarginase) for the treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents [...]
UCLA scientists receive $16.1 million in CIRM grants to advance novel stem-cell based treatments
From left to right: Dr. Gerald Lipshutz, Dr. Zulema Romero Garcia and Dr. Donald Kohn Researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research [...]
Experts Implore FDA to Accept Biomarker Evidence in Ultra-Rare Diseases
By Heather McKenzie See Orignial Story Here Arginase 1 deficiency, or ARG1-D, is a rare, inherited disorder occurring in approximately 1.1 in 1 million live U.S. births. While born with normal function, by the [...]
Loargys® (pegzilarginase) receives positive opinion by the CHMP for treatment of arginase 1 deficiency
Stockholm, October 13, 2023: Immedica today announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending marketing authorization of Loargys® (pegzilarginase) [...]
FDAʼs ʻivory tower thinkingʼ ignores promising biomarkers, says a rare disease CEO
The FDA wouldnʼt review a drug that was 90% effective. In rare diseases, this is too common, said Ultragenyx CEO Dr. Emil Kakkis and a leading rare disease researcher.
Aeglea BioTherapeutics Announces Sale of Pegzilarginase to Immedica Pharma
Aeglea BioTherapeutics Announces Sale of Pegzilarginase to Immedica Pharma Global rights to pegzilarginase in development for Arginase 1 Deficiency sold to Immedica Pharma for $15 million upfront cash proceeds and up to [...]
Arginase 1 Deficiency Foundation Files Petition Urging Aeglea Biotherapeutics to Reinstate Life-Saving Drug Following Abrupt Withdrawal to Clinical Trial Patients in the United States
Arginase 1 Deficiency Foundation Files Petition Urging Aeglea Biotherapeutics to Reinstate Life-Saving Drug Following Abrupt Withdrawal to Clinical Trial Patients in the United States Seattle, Washington, April 21, 2023: The Arginase 1 [...]
Pegzilarginase Clinical Trial Update (January 2023)
Pegzilarginase Clinical Trial Update (January 2023) As Aeglea previously announced, we are in the process of concluding our ongoing pegzilarginase Phase 1 and Phase 3 clinical trials and transitioning those patients [...]