The February 2019 meeting of the Pharmacology and Therapeutics Advisory Committee (PTAC) included review of the November 2018 Rare Disorders Subcommittee meeting recommendations to fund 4 new Medicines.
During its meeting PTAC accepted the high priority recommendations to fund two Te Arai BioFarma products – Carglumic acid (for the treatment of metabolic disorders CPS1 and NAGs) and nitisinone (for the treatment of hereditary type 1 tyrosinemia). PTAC downgraded the Rare Disorders subcommittee recommendation to fund a product for Cystic fibrosis to low priority and made a decision to instead decline funding for a medicine to treat Fabry disease.
Te Arai BioFarma look forward to progressing the only two high priority rare disorder Medicines to being fully PHARMAC funded.
The complete PTAC minutes are available here : Feb 21 and 22 2019