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.
→ Sanofi and Regeneron’s IL-3/IL-14 blockbuster Dupixent has been approved in China for atopic dermatitis. CEO Paul Hudson said that as part of the government’s Healthy China 2030 initiative, the French pharma will seek 25 new approvals by 2025.
→ Sagimet Biosciences released positive Phase II data for its NASH drug TVB-2640. Although the field has struggled despite great investor and industry promise, most recently with the failure of GenFit’s closely-watched Phase III trial, a steady pipeline of earlier stage drugs continue to march along. Sagimet, known as 3-V Biosciences until last August, said the drug’s high dose had a 61% response rate across 30 patients and led to a mean liver fat reduction of 28.2%.