CHMP recommends paediatric oral formulation of Orfadin (nitisinone) for Hereditary Tyrosinaemia type-1 (HT-1)- SOBI

Swedish Orphan Biovitrum AB (Sobi) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the oral suspension formulation and the 20 mg capsule of Orfadin (nitisinone) for the treatment of Hereditary Tyrosinaemia type-1 (HT-1). HT-1 is a rare genetic disease that affects infants and children; it is progressive in nature, may result in liver and kidney failure and can be fatal if untreated. Twenty years ago before pharmacological treatment was available, less than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday.

Treatment with Orfadin combined with dietary restriction of tyrosine and phenylalanine and more widespread newborn screening leading to early diagnosis has dramatically improved outcomes for HT-1 patients. EMA has called for more child-friendly formulations and the new paediatric oral suspension formulation is a response to this call and the needs of the infants and children diagnosed with HT-1 early in life. A paediatric oral suspension of Orfadin, if approved, will facilitate accurate dosing for infants and small children, and contribute to improving quality of life and patient outcomes.