EU approves oral Orfadin (nitisinone) to treat Hereditary Tyrosinaemia type-1 -SOBI

Sobi announced that the European Commission has approved the oral suspension formulation of Orfadin (nitisinone) for the treatment of Hereditary Tyrosinaemia type-1 (HT-1) as of 19 June 2015. HT-1 is a rare genetic disease that affects infants and children. It is progressive and may result in liver and kidney failure and can be fatal if untreated.The oral suspension will be made available to patients throughout Europe in H2 2015.

Before pharmacological treatment was available, fewer 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 new-born screening, leading to early diagnosis have dramatically improved outcomes for HT-1 patients. Orfadin capsules are approved in the EU and several other countries for the treatment of patients with confirmed diagnosis of HT-1 in combination with dietary restriction of tyrosine and phenylalanine.

Comment:The European Medicines Agency had called for more child-friendly formulations and the new oral suspension formulation is response by Sobi to the needs of the infants and children diagnosed with HT-1 early in life. An oral suspension of Orfadin for paediatric use will facilitate accurate dosing for infants and small children, and contribute to improving quality of life for patients and caregivers.