Sobi Canada Inc., a subsidiary of Swedish Orphan Biovitrum AB (publ) (Sobi), announced 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 rare genetic disease that affects infants and children. It is progressive and may result in liver, brain and kidney complications and can be fatal if untreated.

“Sobi now can ensure that children and families impacted by this rare, hereditary disease have ongoing, improved access to this treatment. Canada is home to 10 per cent of the world’s HT-1 population, so this approval represents a significant milestone for this patient community,” says Len Walt, vice president & head of medical affairs, Sobi North America.

Orfadin capsules will now be available in Canada in a wide range of dosing options (2mg, 5mg, 10mg and 20mg). Sobi is the first and only healthcare company to offer nitisinone 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 invest in Canada by developing impactful therapies that meet the needs of patients and healthcare professionals.”

Twenty years ago, before pharmacological treatment was available, fewer than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday. Today, treatment with Orfadin, combined with dietary restriction of tyrosine and phenylalanine and more widespread newborn screening leading to early diagnosis, has greatly improved outcomes for HT-1 patients.

Orfadin is also approved in the US and Europe and is a proprietary product developed and made available globally by Sobi.

People with hereditary tyrosinaemia type-1 (HT-1) have problems breaking down an amino acid called tyrosine. Toxic by-products are formed and accumulate in the body, which can cause liver, renal and neurological complications. In the most common form of the disease, symptoms arise within the first six months of the child's life. Approximately 1,000 persons worldwide are identified as living with HT-1 today, 10 per cent of whom live in Canada. Orfadin (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic tyrosine byproducts in the body. Patients must maintain a special diet in combination with Orfadin treatment as tyrosine is not adequately broken down.