Swedish Orphan Biovitrum (Sobi) has received approval from Health Canada for an oral suspension formulation of Orfadin (nitisinone) to treat patients with hereditary tyrosinemia type 1 (HT-1).
Patients suffering from HT-1, a rare genetic disorder, have problems breaking down an amino acid called tyrosine.
This results in the formation of toxic by-products that accumulate in the body and can cause liver, renal and neurological complications, and in most cases is fatal if left untreated.
Developed by Sobi, Orfadin is the first liquid formulation nitisinone product approved in Canada and can meet the needs of small children and patients who have difficulty in swallowing capsules.
It is the first therapy approved in multiple countries worldwide for use in combination with dietary restriction of tyrosine and phenylalanine for the treatment of HT-1.
Both Orfadin oral suspension and Orfadin 20mg capsules are currently available in Canada, in addition to the original 2mg, 5mg and 10mg capsules.
Sobi Canada vice-president and general manager Bob McLay said: “The introduction of new nitisinone dosing options, such as Orfadin oral suspension, is the result of Sobi listening to feedback from patients and caregivers and continuing to innovate to meet their needs.
“In various markets around the world where Sobi has already introduced multiple innovative Orfadin formulations such as Orfadin oral suspension and Orfadin 20mg capsules, we have seen patients increasingly choose these dosing options over the first generation capsule, so we are excited to offer a choice to patients in Canada as well.”
The original approval of the Sobi treatment in Canada was based on a multinational, uncontrolled, open-label trial carried out on a total of 291 patients with HT-1.