36. Givlaari


Active ingredient: givosiran
Disease: acute hepatic porphyria
Peak sales estimate: $500 million
Approved: Nov. 20, 2019
Company: Alnylam 

The scoop: Facing a major challenge to its RNAi therapy Onpattro, Alnylam afforded itself some breathing room with the approval of its second such therapy only a year later. The FDA approved Givlaari (givosiran) in November to treat acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules. In an effort to help insurers cover the pricey $575,000 annual per-patient treatment cost, Alnylam touted its unusual “prevalence-based” reimbursement strategy that would offer significant rebates to insurers if patient numbers outshout expectations. The FDA based its approval off results from a phase 3 trial showing Givlaari beat placebo at reducing the annualized rate of composite porphyria attacks including those requiring hospitalization, an urgent healthcare visit, or an administration of hemin, which is used to treat porphyria attacks. –– Kyle Blankenship



36. Givlaari

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