Our treatment

Orfadin is the first FDA-approved therapy of HT-1 along with a tyrosine and phenylalanine-restricted diet. But our product innovation extends beyond Orfadin’s medicinal properties. We are constantly striving to make dosing and administration as simple and personalized as possible.

One dose a day

Orfadin is the first and only FDA-approved, once-daily dosing option for 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. All dosages, including the oral suspension, may only need to be taken once a day.

Room temperature storage

Orfadin can be stored at room temperature for more than a month, so you can take it on sleepovers, camping, road trips, and long vacations.

• Orfadin capsules can be safely stored at room temperature (77°F/25°C or lower) for up to 45 days.

• Orfadin oral suspension can be safely stored at room temperature (77°F/25°C or lower) for up to 60 days after opening.

Flexible dosing options

When it comes to HT-1 treatment, there is no "one size fits all." We offer a range of dosing options so you and your doctor can tailor your treatment plan.

• Orfadin is the only liquid oral suspension nitisinone that comes ready to use, for administering treatment to infants and young children.

• Orfadin is available in 2-mg, 5-mg, 10-mg, and 20-mg capsules so the treatment plan can be tailored to the patient.

• Our 20-mg option can reduce the number of capsules you need to take.

Organization made easy

Our easy-to-use blister pack may help you keep track of the pills you’ve already taken, and the ones you still need to take.