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Fresh off its first drug approval, Alnylam announced positive interim data from a trial of another gene-silencing treatment on Thursday and said it hopes to file with the Food and Drug Administration for accelerated approval of the drug, givosiran, likely by the end of the year.

The Cambridge, Mass.-based company did not release any data Thursday, but said in a statement that an interim analysis of a Phase 3 trial of givosiran showed it successfully lowered levels of a chemical that regulators have said they will consider a surrogate endpoint to gauge the drug’s effectiveness.


Givosiran is being tested as a treatment for acute hepatic porphyrias, a rare genetic disease that causes abdominal issues that can be fatal. The primary endpoint of the study is the rate of porphyria attacks that require medical attention. But for the interim analysis, which looked at 43 patients, the company focused on the reduction of a chemical in the urine called ALA.

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