Givosiran was approved by the US FDA for the treatment of acute hepatic porphyria, a rare generic blood disorder, nearly three months ahead of the action date.

Brazil has established an accelerated pathway for approval of drugs for ultra-rare diseases, encouraging development activity – but the definition of qualifying drugs is narrow. Expansion to include more treatments could boost clinical trials activity and companies’ overall development presence.

A new publication offers up proof of concept that the genetic signatures of microorganisms found in blood and tumor tissue could improve precision medicine cancer diagnostics. The study also bolsters the notion that targeting the microbiome could lead to innovative cancer therapies.