Sobi’s Orfadin® (nitisinone) Receives Positive Reimbursement Recommendation for the Treatment of HT-1 from CADTH Canadian Drug Expert Committee

Published: Feb 27, 2018

OAKVILLE, Ontario--(BUSINESS WIRE)-- Sobi Canada Inc., a pioneering international biopharmaceutical company dedicated to rare and difficult-to-treat diseases, announced today that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued a conditional positive reimbursement recommendation for Orfadin® (nitisinone) in the treatment of adult and pediatric patients with hereditary tyrosinemia type-1 in combination with dietary restriction of tyrosine and phenylalanine, if certain conditions and criteria are met. This recommendation represents an important step towards access to Orfadin for Canadian patients. Sobi intends to continue taking the necessary steps to ensure Orfadin remains accessible to Canadian patients.

Orfadin is approved in multiple countries globally for use in combination with dietary restriction of tyrosine and phenylalanine in the treatment of HT-1. HT-1 is a rare genetic disorder that may result in liver, renal and neurological complications and in most cases is fatal if untreated. Sobi provided Orfadin to Canadian HT-1 patients for decades through Health Canada’s Special Access Programme; the company established a Canadian affiliate in 2015 and gained Health Canada approval for Orfadin in late 2016. Sobi currently provides a range of Orfadin dosing options in Canada including 2mg, 5mg, 10mg, and 20mg capsules, as well as a 4mg/mL oral suspension.

“Since establishing Sobi’s Canadian affiliate in 2015 our team has been committed to securing sustainable access to Orfadin for the Canadian HT-1 community. Sustainable access means that a drug is approved and reimbursed in a given country, which helps assure consistent supply and is particularly important for drugs like Orfadin, where daily administration may be critical to the patients’ health throughout their entire life,” said Bob McLay, Vice President and General Manager of Sobi Canada Inc. “This positive reimbursement recommendation for Orfadin is a step towards public drug plan reimbursement, and hence a step towards ensuring that all Canadian HT-1 patients have sustainable access to Orfadin.”

The positive CDR recommendation is based on the condition that Orfadin is prescribed by a physician with experience in the diagnosis and management of HT-1 and that pricing negotiations be successfully completed. The recommendation recognizes an association between nitisinone plus dietary treatment and improved survival, versus dietary treatment alone. Nitisinone was also associated with reduced risk of liver failure, fewer liver transplantation requirements, lower risk of hepatocellular carcinoma, fewer porphyric crises, and reduced acute complications of HT-1 versus dietary treatment alone. The report also considered patient feedback regarding the importance of universal accessibility and interruption-free availability of nitisinone during any transition to the Public Drug Plans in Canada and throughout an HT-1 patient’s lifetime. CDEC also noted that jurisdictions that do not perform newborn screening for HT-1 may wish to also consider the cost-effectiveness of introducing such screening, thereby facilitating early identification of eligible patients.

CADTH is an independent, not-for-profit organization responsible for providing Canada’s health care decision-makers with objective evidence to help make informed decisions about the optimal use of drugs, medical devices, diagnostics, and procedures in the health care system.

About hereditary tyrosinemia (HT-1)

People with hereditary tyrosinemia 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 most cases HT-1 is fatal if untreated. Approximately 1,000 persons worldwide are identified as living with HT-1 today. Roughly 10 percent of those patients reside in Canada.

About Orfadin®

Orfadin® (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic tyrosine by-products in the body. Patients must maintain a special diet in combination with Orfadin treatment as tyrosine is not adequately broken down. Orfadin is a proprietary product and is developed and made available globally by Sobi. For full European prescribing information, please visit the EMA website. For full US prescribing information please see Full Canadian prescribing information can be found on the Sobi North America website.

About Sobi™

Sobi™ is an international specialty healthcare company dedicated to rare diseases. Our vision is to be recognized as a global leader in providing access to innovative treatments that make a significant difference for individuals with rare diseases.

The product portfolio is primarily focused on treatments in Hemophilia and Specialty Care. Partnering in the development and commercialization of products in specialty care is a key element of our strategy. Sobi has pioneered in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2017, Sobi had total revenues of SEK 6.5 billion and approximately 850 employees. The share (STO:SOBI) is listed on Nasdaq Stockholm. More information is available at and



Trista Morrison, 781-810-0490


Source: Sobi

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