Oportunitat de mercat
There are approximately 12,000 patients affected with Cystinuria in the US, about 20,000 in Europe, and a worldwide prevalence of 1 in 7,000 people. Given this market size, it could be considered as an “orphan” indication. Historically, the primary therapy focused on Cystinuria was Penicillamine, marketed by Valeant. However, nearly a third of people are allergic to it. Consequently, this opened the door to Tiopronin, which is marketed by Retrophin Inc, and showed fewer side effects. According to RTRX, it has identified 3,000 patients with Cystinuria (currently treating 425). At pricing of ~US$80,000 per patient per year, 3,000 patients would represent a market opportunity of US$240 million per year.