Alnylam goes 3-for-3 in rare disease trials
Alnylam Pharmaceuticals said yesterday that its treatment for a rare liver disease met its goals in a pivotal trial, marking the company’s third straight clinical success and setting the stage for FDA approval.
As STAT’s Kate Sheridan reports, Alnylam’s lumasiran, an RNAi therapy, is meant to treat primary hyperoxaluria type 1, or PH1, which results from an excess of a chemical called oxalate. In a clinical study, the drug reduced the amount of oxalate in patients’ urine, a result the company believes will translate to relief from PH1’s painful symptoms.
But competition is on the way from Dicerna Pharmaceuticals, Alnylam’s former courtroom enemy. DCR-PHXC, a treatment that targets PH1 through a different pathway, is between nine and 12 months behind lumasiran.
Read more.
As STAT’s Kate Sheridan reports, Alnylam’s lumasiran, an RNAi therapy, is meant to treat primary hyperoxaluria type 1, or PH1, which results from an excess of a chemical called oxalate. In a clinical study, the drug reduced the amount of oxalate in patients’ urine, a result the company believes will translate to relief from PH1’s painful symptoms.
But competition is on the way from Dicerna Pharmaceuticals, Alnylam’s former courtroom enemy. DCR-PHXC, a treatment that targets PH1 through a different pathway, is between nine and 12 months behind lumasiran.
Read more.
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