Health Canada initiates approval application review for Sobi’s HT-1 treatment

14 July 2016 (Last Updated July 14th, 2016 18:30)

Health Canada has initiated a review of the application for approval of healthcare company Swedish Orphan Biovitrum’s (Sobi) Orfadin (nitisinone) capsules to treat hereditary tyrosinaemia type-I (HT-1).

Health Canada has initiated a review of the application for approval of healthcare company Swedish Orphan Biovitrum’s (Sobi) Orfadin (nitisinone) capsules to treat hereditary tyrosinaemia type-I (HT-1).

HT-1 is a rare but progressive genetic disease that causes liver and kidney problems in infants and children and can be fatal if left untreated.

People affected with HT-1 face problems in breaking down an amino acid called tyrosine, which results in the formation of toxic by-products that accumulate in the body, causing liver, renal and neurological issues.

Orfadin (nitisinone) helps block the breakdown of tyrosine, thereby reducing the amount of toxic tyrosine by-products in the person’s body.

"If approved, it will be a major step in ensuring that HT-1 patients throughout Canada could have sustainable access to Orfadin treatment going forward."

Sobi core poducts global medical affairs head Michael Yeh said: “We are very pleased that the review process for Orfadin capsules in Canada has started.

“Canada and Quebec is home to 10% of the world’s HT-1 population and has been a priority for Sobi since we took direct responsibility for Orfadin in the region.

“If approved, it will be a major step in ensuring that HT-1 patients throughout Canada could have sustainable access to Orfadin treatment going forward.”

Orfadin (nitisinone) is approved in the US and Europe to treat patients with HT-1, along with dietary restriction of tyrosine and phenylalanine.

Once approved in Canada, the range of dosing alternatives, 2mg, 5mg, 10mg, and 20mg capsules, will be able to offer personalised treatment for HT-1 patients.