Posted by Michael Wonder on 11 Feb 2020
SMC approves first two ‘ultra-orphan’ pathway meds
11 February 2020 - The Scottish Medicines Consortium has released its February guidance, advising six new medicines to NHS Scotland.
Among the decisions are the first two medicines assessed within the new “ultra-orphan pathway”, a new approach introduced earlier this year for drugs that are intended for extremely rare conditions.
In the SMC’s latest meeting, Novartis’ Luxturna (voretigene neparvovec) and Kyowa Kirin’s Crysvita (burosumab) were the two new ultra-orphan drugs to be instated by the body, a one-time therapy for an extremely rare type of inherited retinal dystrophy and a treatment for X-linked hypophosphataemia, respectively.
