The European Commission has approved an oral suspension formulation of Swedish Orphan Biovitrum’s (Sobi) Orfadin (nitisinone).
The orphan drug treats Hereditary Tyrosinaemia type-1 (HT-1), a rare genetic and potentially fatal condition that affects infants and children and can result in liver and kidney failure if untreated, according to PMLiVE.
“The European Medicines Agency has called for more child-friendly formulations and the new oral suspension formulation is a demonstration of Sobi’s commitment to the needs of the infants and children diagnosed with HT-1 early in life,” says Sobi’s chief medical officer Birgitte Volck. “An oral suspension of Orfadin for pediatric use will facilitate accurate dosing for infants and small children, and contribute to improving quality of life for patients and caregivers.”
Orfadin blocks the breakdown of tyrosine to reduce the amount of toxic by-products in the body that can lead to liver failure, renal dysfunction and neurological complications.
The drug was first approved in 2005 in capsule form and is used in conjunction with a modified diet that limits tyrosine and phenylalanine. Approximately 1,000 individuals have been identified as living with the disease.