Health Canada approves Orfadin capsules for the treatment of hereditary tyrosinaemia type-1 (HT-1)

Swedish Orphan Biovitrum AB (publ) (Sobi) today announces that Health Canada has approved Orfadin (nitisinone) capsules for the treatment of hereditary tyrosinaemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

HT-1 is a progressive, rare genetic disease that may result in liver and kidney complications and in most cases fatal if untreated.

In the most common form of the disease, symptoms arise within the first six months of the child's life.

Orfadin capsules will now be available in Canada in a wide range of dosing options; 2mg, 5mg, 10mg and 20mg. Sobi is the first company to offer Orfadin® capsules at the 20mg dosage option, which may allow patients to take fewer pills per day.

“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,” says Bob McLay, Vice President, General Manager, Sobi Canada Inc.

“This approval is an exciting moment for Sobi, as we continue to develop impactful therapies that meet the needs of patients and healthcare professionals”.

Before Orfadin became available, the survival rate in HT-1 was 29% after two years for children who developed symptoms before two months of age.[1]After the introduction of Orfadin, the survival rate is 93% after two years in patients with treatment initiation before two months of age.[2]

Today, treatment with Orfadin as an adjunct to dietary restriction as well as early diagnosis and treatment initiation have dramatically improved outcomes for HT-1 patients and their families.

Orfadin is also approved in the US and Europe for the treatment of patients with confirmed diagnosis of HT-1 in combination with dietary restriction of tyrosine and phenylalanine.

  1. van Spronsen FJ, Thomasse Y, Smit GP, et al. Hepatology. 1994;20(5):1187-1191
  2. Orfadin Product monograph 12/12/2016 (Submission Control No: 193226)

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