Promises made, promises kept: Alnylam CEO John Maraganore adds 3rd straight positive pivotal as he aims at another new drug OK
Alnylam CEO John Maraganore has been keeping his promises this year.
Following the historic first OK for an RNAi drug last year, the crew at Alnylam pushed through a second approval recently. And now its third straight pivotal success sets up another likely near-term OK for an ultra-rare disease drug, with more promising additions looming in the near future.
The pivotal came through for lumasiran, their RNAi therapy for the few thousand cases of primary hyperoxaluria type 1, or PH1, that occur in the US every year. The headline-only results: The study met the primary and secondary endpoints — tracking 24-hour urinary oxalate excretion — and investigators say they found an “encouraging” safety and tolerability profile.
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