Novartis' PI3K drug, already approved in breast cancer, nabs a speedy OK to treat ultra-rare condition
Half a year after Novartis showcased the potential of its PI3K inhibitor alpelisib among a group of young patients with an ultra-rare condition, the FDA has greenlighted the drug’s use based solely on real-world evidence.
Branded Vijoice, it is the first and only treatment aimed at the root cause of PIK3CA-related overgrowth spectrum (PROS), according to Novartis, a disease estimated to afflict 14 people per million.
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