The European Commission has approved an oral suspension formulation of Swedish Orphan Biovitrum’s (Sobi) Orfadin (nitisinone).
The orphan drug treats the rare genetic disease Hereditary Tyrosinaemia type-1 (HT-1), a potentially fatal condition that affects infants and children and can result in liver and kidney failure if untreated.
Sobi’s chief medical officer Birgitte Volck said: “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.
“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.”
Orfadin works by blocking the breakdown of tyrosine to reduce the amount of toxic by-products in the body that can cause liver failure, renal dysfunction and neurological complications.
The drug was first approved in 2005 in a capsule formulation, in combination with dietary restriction of tyrosine and phenylalanine, to treat HT-1, which just 1,000 persons have been identified as living with today.
Last year sales of Orfadin rose by almost 50% to bring in SEK 547.9m and Sobi plans to launch the drug’s new formulation in the second half of this year.
