Dear Arginase 1 Deficiency Community & Families,
If you have not seen already, we are excited to share that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Loargys® (pegzilarginase-nbln), a prescription medicine used to treat high levels of arginine in the blood of adults and children 2 years and older with Arginase 1 Deficiency (ARG1-D), along with limiting the amount of protein in their diet.
We understand that this is a historic and long-awaited day for the ARG1-D community, and we’re appreciative of the community’s patience and partnership throughout this journey.
We extend our deepest gratitude to the families and healthcare professionals whose participation and contributions to clinical trials helped make this milestone possible. Your commitment and sacrifice helped to make the first and only therapy proven to lower arginine available.
Important Notes and Next Steps:
- Although LOARGYS has been approved, it is not yet available. We anticipate the medication to be available in April 2026.
- We will remain in close communication with the patient advocacy organizations supporting ARG1-D and Urea Cycle Disorders, as well as healthcare providers, and will share updates as more information becomes available.
- Please contact your healthcare provider(s) if you have any questions about the medication.
To support access to treatment, Immedica is launching There for Rare, a patient support program providing nonmedical education to help eligible individuals prescribed LOARGYS navigate each stage of the treatment journey, including financial assistance options.
If you have any questions, please reach out to our Patient Advocacy team directly at
tom.damato@immedica.com or bj.viau@immedica.com.
Sincerely,
Dan Camardo
President, Immedica North America