Alnylam Going After Alexion's Soliris In PNH, Eyes Roche In Hemophilia
This article was originally published in The Pink Sheet Daily
At an investor day, the RNAi firm outlined Phase I programs in paroxysmal nocturnal hemoglobinuria, hemophilia and hepatic porphyria, and said it is using a de-risked drug development methodology.
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The company expects to complete a rolling NDA submission for its second RNAi therapeutic in mid-2019, and sees an opportunity to target severe and more moderate patients with acute hepatic porphyria.
The RNA interference-focused biotech will bring the rest of its pipeline forward with subcutaneous technology now that it has been validated in human studies, but the company will continue to pursue approval of its lead candidate as an infusion product.
The median review time for novel agents at CDER hasn’t changed in five years. Our infographic offers a look at the tortoises and hares (all of them winners because they got approved).