FDA nod for reduced Orfadin dosing

Swedish Orphan Biovitrum (STO: SOBI) has received approval from the US Food and Drug Administration (FDA) for a reduced dosing frequency for Orfadin (nitisinone) from twice daily to once daily for the treatment of hereditary tyrosinaemia type 1 (HT-1).

This approval is based on the results of a clinical study in 16 people with the rare condition, comparing a four-week once-daily and four week twice daily dosing regimen.

The reduced dosing frequency is approved for use in patients five years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of four weeks on a stable dosage of nitisinone. A once-daily dosing option was also approved by the European Commission in the beginning of 2017.