Japan Grants Orphan Drug Designation to Pegzilarginase for Arginase 1 Deficiency – Immedica

Immedica announces that Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to pegzilarginase for the treatment of arginase 1 deficiency (ARG1-D), a rare inherited metabolic disorder.

“Receiving Orphan Drug Designation for pegzilarginase in Japan is an important step in our commitment to bringing innovative treatments to patients living with rare diseases worldwide. We look forward to continuing our engagement with the Japanese regulatory authority as we advance our plans to make pegzilarginase available to patients with ARG1-D in Japan.” – Anders Edvell, CEO of Immedica.

Following the Orphan Drug Designation, Immedica is working towards the submission of a Japanese New Drug Application (J-NDA) for pegzilarginase. In Japan, products granted Orphan Drug Designation are eligible for a priority review process, which may result in a shorter regulatory review timeline compared with standard applications.