Ultragenyx’s ultra-expensive ultra-rare disease drug earns second nod; Another round of promising NASH data
→ Ultragenyx’s Crysvita has scored its second FDA approval, earning the nod to treat bone softening in patients with the ultra-rare disease known as tumor-induced osteomalacia. First approved in 2018 for a different ultra-rare indication, x-linked hypophosphatemia, Crysvita quickly earned watchdog scorn for its $200,000 per year price tag. Despite hopes, though, that the price would bring blockbuster sales, it earned only $87.3 million last year and $28.8 million in Q1 2020. The drug is also marketed by Japanese biotech Kyowa Kirin.
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