Posted by Michael Wonder on 10 Sep 2017
Sobi receives approval from the FDA for once-daily dosing frequency of Orfadin for the treatment of HT-1
5 September 2017 - Sobi has received approval from the U.S. FDA for a reduced dosing frequency for Orfadin (nitisinone) from twice daily to once daily in patients 5 years of age and older.
The approval is based on the results of a clinical study in 16 people with hereditary tyrosinaemia type 1 (HT-1), comparing a four week once-daily and four week twice daily dosing regimen.
Orfadin is approved in combination with dietary restriction of tyrosine and phenylalanine for people with HT-1, a rare genetic disorder that can cause liver, renal and neurological complications. The reduced dosing frequency is approved for use in patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage of nitisinone. A once-daily dosing option was also approved by the European Commission in the beginning of 2017.
