27 February 2018 - Sobi announced today that the CADTH Canadian Drug Expert Committee has issued a conditional positive reimbursement recommendation for Orfadin (nitisinone) in the treatment of adult and paediatric 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.

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.

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