Arginase 1 Deficiency News
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 [...]
Aeglea BioTherapeutics Submits BLA to FDA for Pegzilarginase for the Treatment of Arginase 1 Deficiency
Aeglea BioTherapeutics Submits BLA to FDA for Pegzilarginase for the Treatment of Arginase 1 Deficiency SEE ORIGINAL STORY HERE BLA submission provides FDA with all pegzilarginase program data to review in [...]
Redacted FDA Listening session
REDACTED - FDA Family-Led Listening Session Report On June 10, 2021, a group of patients and families affected by Arginase 1 Deficiency (ARG1-D) participated in a one-hour virtual listening session with [...]
Caregivers’ Experiences Survey
We’re proud to partner with the Child Neurology Foundation to conduct a survey to understand caregivers’ experiences on their journey in child neurology. Results will be shared with advocates and health care [...]