Arginase 1 Deficiency News

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 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 Seattle, Washington, April 21, 2023: The Arginase 1 [...]

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 SEE ORIGINAL STORY HERE BLA submission provides FDA with all pegzilarginase program data to review in [...]

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 [...]

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 [...]

PEGZILARGINASE Trial Discussion. Amazing presentation with Dr. Diaz, Dr. Burton and our own Tanja Brandt discuss Arginase 1 Deficiency, the Phase 2 and Phase 3 trial. Tanja discusses the day to day [...]

I am excited to share with you that today we announced our Phase 3 PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints) study achieved its primary endpoint with a statistically significant [...]

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 officials from [...]

Aeglea BioTherapeutics and Immedica Announce Commercialization Agreement for Pegzilarginase for the Treatment of Arginase 1 Deficiency in Europe and Middle East Published: Mar 22, 2021 Original Story HERE AUSTIN, Texas and STOCKHOLM, [...]

Aeglea BioTherapeutics Launches THINK ARGININE™, a Disease Education Initiative to Improve Awareness and Diagnosis of Arginase 1 Deficiency 05/06/2021 Original Story HERE Includes Sponsored Diagnostic Testing and Services for Patients with Suspected [...]