Rare disease group Swedish Orphan Biovitrum (Sobi) says it has gained NHS England reimbursement for Ravicti for long-term management of urea-cycle disorders (UCDs) in adults and children.
This drug is the first liquid formulation of phenylbutyrate to be offered to patients whose condition cannot be controlled by diet via commissioning centres across England.
A UCD is a rare, inherited genetic condition where patients are unable to get rid of waste nitrogen from their bodies. This then accumulates in the form of ammonia and may lead to irreversible brain damage, coma and/or death if untreated.
Ravicti reduces levels of ammonia in the body both in the short and long term, offering an alternative to current treatment, which can be difficult for patients – particularly children - to adhere to because of an unpleasant taste and the high number of tablets needed.
Glycerol phenylbutyrate, a pre-prodrug of phenylacetate that is released gradually following administration, is nearly odourless and tasteless, requires minimal preparation and offers easy administration, the firm noted.
“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,” noted Joanne Taylor, acting chief executive, Metabolic Support UK.
“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.”
The drug was recently also accepted by the Scottish Medicines Consortium and Irish authorities, meaning that the drug will be available to NHS patients ariss England, Scotland and Ireland.