Patients in Quebec with the rare, life-threatening disease HT-1 (Hereditary Tyrosinemia type 1) received a boost today with the announcement that INESSS, the Institut National d’Excellence en Santé et Services Sociaux Quebec, is making its first ever recommendation for an HT-1 treatment, Nitisinone Tablets.
HT-1 is a rare genetic disease that is unusually common in Quebec, particularly in the Saguenay-Lac-Saint-Jean region, affecting children from birth. Untreated, it leads to serious health consequences that can include childhood liver transplants or early death.
Cycle Pharmaceutical’s Nitisinone Tablets, approved by Health Canada in November 2016, is the first HT-1 treatment to pass through INESSS’ rigorous evaluation. The INESSS’ Scientific Committee have agreed unanimously that Cycle’s Nitisinone Tables satisfies all therapeutic value criteria including the prevention of deaths and liver transplants, as well as neurological crises and hospitalizations. There is no approved HT-1 treatment currently on the Quebec List of Medications. This recommendation represents a significant step forward to provide a new level of treatment access for HT-1 patients