Health Canada has approved Swedish Orphan Biovitrum’s (Sobi) Orfadin (nitisinone) capsules to treat hereditary tyrosinaemia type 1 (HT-1) disease in combination with a dietary restriction of tyrosine and phenylalanine.
HT-1 may result in liver and kidney complications and in most cases could prove fatal if not treated.
Symptoms arise within the initial six months of the child's life in the common form of the disease.
Following the approval, Orfadin capsules will now be available in Canada in 2mg, 5mg, 10mg and 20mg dosing options.
The 20mg dosage option will allow patients to take fewer pills per day.
Sobi Canada vice-president and general manager Bob McLay said: “Sobi has been committed to supporting the HT-1 community globally for more than two decades, and we were the first to develop this treatment and make it available in Canada through Health Canada’s Special Access Programme.
“This approval is an exciting moment for Sobi, as we continue to develop impactful therapies that meet the needs of patients and healthcare professionals.”
Prior to the availability of Orfadin capsules, the survival rate in HT-1 was 29% after two years for children who developed symptoms before two months of age.
After the introduction of the capsules, the survival rate increased to 93%.
Orfadin is also approved in the US and Europe to treat patients with confirmed diagnosis of HT-1.
People with HT-1 have problems breaking down an amino acid called tyrosine and about 1,000 people worldwide are identified as living with HT-1.
Orfadin (nitisinone) works by blocking the breakdown of tyrosine and therefore reducing the amount of toxic tyrosine by-products in the body.