NHS England and the Scottish Medicines Consortium agree to reimburse Ravicti® (glycerol phenylbutyrate) for the management of Urea Cycle Disorders (UCDs)
Ravicti is the first liquid formulation of phenylbutyrate, which is virtually tasteless and odourless. This treatment, a more palatable alternative to sodium phenylbutyrate will be provided via specialised metabolic commissioning centres across England and Scotland for the long-term management Urea Cycle Disorders (UCDs) in adults and children from the age of two months who cannot be managed by changes in their diet alone. Unlike other phenylbutyrate products, which are in powder or tablet form, liquid glycerol phenylbutyrate may be taken by mouth via an oral dosing syringe.
UCDs are a group of very rare, serious, life-threatening, inherited defects of the urea cycle, the biological process responsible for converting ammonia to urea, a waste product that is excreted in urine. Failure to convert ammonia in affected patients leads to the accumulation of toxic ammonia levels in the blood and brain. High ammonia levels can cause coma and irreparable brain damage, which may result in long-term complications such as cognitive impairment, seizures, cerebral palsy and death if not properly managed. UCDs affect patients of all age groups, with some exhibiting symptoms shortly after birth and others presenting during adulthood. UCD patients are treated and monitored from the time of onset onwards.
Joanne Taylor, Acting Chief Executive, Metabolic Support UK – “UCDs are chronic conditions that patients must manage throughout the course of their lives. Therefore, any management strategy that might ease the burden of regular treatment could be of great benefit to them. The palatability and ease of administration of glycerol phenylbutyrate could mean that UCD patients are more likely to be compliant. This may result in fewer hospital admissions for acute hyperammonaemic episodes and could therefore improve long-term patient outcomes.”
If any UCD patient, currently taking sodium phenylbutyrate would like to access Ravicti as an alternative treatment, they are advised to discuss this with their metabolic teams.