Swedish Orphan Biovitrum (Sobi) — a pharmaceutical company that specialises in rare diseases — has secured NHS England reimbursement for its medicine for the management of urea cycle disorders (UCDs), Ravicti (glycerol phenylbutyrate).
NHS money
The medicine is used for the long-term management of UCDs in adults and children (from the age of two-months old) who are unable to be managed through dietary changes alone. It is the first liquid formulation of phenylbutyrate to be offered to patients via commissioning centres across England. Other phenylbutyrate products are in powder or tablet form so in this liquid formulation it will possible for the medicine to be taken by an oral dosing syringe.
“The reimbursement of glycerol phenylbutyrate means that we have a new management tool that may benefit our UCD patients and help to advance their care,” stated Dr Robin Lachmann, consultant in Inherited Metabolic Diseases, University College London Hospitals. “UCD patients need to maintain normal levels of blood ammonia to reduce the risk of acute hyperammonaemic episodes and associated chronic complications. At present, protein restriction diets and nitrogen scavenging treatments are the main methods used to decrease and remove excess ammonia, respectively. Amino acid supplementation is also used to manage those patients on low protein diets.”
“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,” added 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.”