Immedica and OrphanPacific announce approval of Ravicti® (glycerol phenylbutyrate) in Japan for the treatment of UCDs
Stockholm, December 22, 2025 - Immedica and its partner OrphanPacific, Inc. announce that the Japanese Ministry of Health, Labour and Welfare (MHLW) has granted marketing authorization for Ravicti® (glycerol phenylbutyrate) for use as adjunctive therapy for the chronic management of patients with urea cycle disorders (UCDs).
Ravicti is a nitrogen scavenger developed for long-term management of UCD. In the pivotal clinical study, as well as in clinical studies conducted in Japanese patients, glycerol phenylbutyrate improved and sustained ammonia control with a favorable safety profile. These results were consistent with international clinical data and support its role as a treatment option for patients requiring ongoing nitrogen scavenger therapy.
“We are very pleased to announce the approval of Ravicti in Japan, which represents an important milestone in our commitment to improving global access to treatments for patients with rare diseases”, says Anders Edvell, CEO of Immedica Pharma AB.
Megumi Hara, President and Representative of OrphanPacific, Inc., comments:
“We are pleased that Ravicti has received approval in Japan. Long-term metabolic control is essential for patients living with UCDs, and we hope that this approval will contribute to improved disease management, quality of life and access to treatment for patients and their families.”
Immedica works with experienced local partners to ensure access to treatments for rare diseases in different markets. In Japan, OrphanPacific is responsible for the regulatory approval, commercialization, and local activities related to Ravicti.
About Urea Cycle Disorders (UCDs)
Urea cycle disorders are a group of metabolic diseases that affect a specific enzyme or transporter in the urea cycle leading to elevated ammonia or glutamine levels in the circulation. Symptoms of the disorder can begin at any age, where more severe defects are seen with an onset of the disease early in life. UCD patients may experience episodes, called hyperammonemic crises, when ammonia levels in the blood become excessively high, which can result in irreversible brain damage, coma, or death. Beyond hyperammonemic crises there are also more subtle symptoms including vomiting, refusal to feed, irritability, muscular hypotonia as well as delayed motor and psychointellectual development. As a group, these disorders occur in 1 in 35,000 newborns.
About Ravicti® (glycerol phenylbutyrate)
Ravicti is a medicine used to treat patients of all ages with UCDs, including deficiencies of carbamoyl phosphate synthetase I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
The medicine is used to reduce the amount of ammonia in the blood in order to reduce the risk of neurological consequences.
About Immedica
Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology and specialty care. Immedica was founded in 2018 and employs today around 160 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo.
For more information visit www.immedica.com.
Immedica contact:
Linda Holmström
Head of Communication
[email protected]
