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Advancing the care for patients with urea cycle disorders

Sobi has announced that the company has gained reimbursement for Ravicti®, a new therapy option for the treatment of patients with urea cycle disorders (UCDs) in several EU member states and EEA-countries.

Sweden, Denmark, Austria and Germany are the first countries to launch, followed by UK, Spain and the Netherlands.

We are very pleased that Ravicti is gaining reimbursement in countries across Europe for patients with Urea Cycle Disorders”, said Norbert Oppitz, Head of Specialty Care at Sobi. “The reimbursement enables us to provide the medical community and patients with a sustainable access to the new drug.”

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In Europe, Ravicti (glycerol phenylbutyrate) is indicated for chronic management of UCDs in adults and children from the age of two months, when the disease cannot be managed by dietary protein restriction and/or amino acid supplementation alone. UCDs are inborn errors of metabolism comprising a group of inherited deficiencies of one of the enzymes or transporters involved in the urea cycle, which converts ammonia to urea.

UCDs are very rare, serious and life-threatening disorders. Absence or severe dysfunction of the enzymes or transporters involved results in an accumulation of toxic levels of ammonia in the blood and brain of the patients. Elevated ammonia levels can cause coma and irreparable brain damage, potentially resulting in cognitive impairment, seizures, cerebral palsy, and even death if untreated.

For patients with UCD, early treatment and adequate ammonia control are critical for maintaining the intellectual function, preventing neurologic damage and reducing the frequency of hyperammonaemic crises.

There is a significant medical need for a more effective treatment of UCDs”, said Armin Reininger, Head of Medical & Scientific Affairs at Sobi. “We believe that the introduction of Ravicti offers a new option to manage ammonia levels and will contribute to further advancing the care of patients with UCDs.”