Urea Cycle Disorders (UCD) are rare genetic conditions caused by an enzyme deficiency. The urea cycle is responsible for removing excess levels of ammonia from the body. Individuals with UCD deal with elevated ammonia levels. The condition can result in brain damage, coma, and even death. It affects individuals of all ages, but up until now, Raviciti (glycerol phenylbutyrate), one of the treatments for the condition wasn’t approved for people under two months of age.
But Horizon Pharma has just announced that Ravicti is now FDA approved for all individuals, regardless of age.
Ravicti is an oral liquid used in combination with protein restriction to help UCD patients maintain ammonia levels. In a clinical trial of the drug for newborns, Ravicti showed safety and efficacy. Patients in the study were able to maintain more stable levels of ammonia during the trial than they were before beginning Ravicti. This trial included patients who were treatment-naive and those who had tried other medications before Ravicti.
Since Ravicti is an oral medication, it’s naturally easier to administer to newborns, meaning it may be exceptionally beneficial for that population of patients.
This new approval will give more patients more options when it comes to treatment of this rare condition. The newborn population is already an especially vulnerable one, and this treatment has the potential to improve outcomes for babies with UCD.
The scientific community is also working to improve early diagnosis through newborn screening. The hope is that through earlier diagnosis and the creation and approval of more treatment options such as Ravicti, we will be able to improve the lives of those living with Urea Cycle Disorders.
You can read more about this new approval and Horizon Pharma’s work here.