Alnylam preps for another PhIII RNAi study, ‘breakthrough’ status in hand
Touting early-stage evidence that its RNAi drug for rare cases of acute hepatic porphyria had begun to demonstrate its promise in the clinic, Alnylam today picked up the FDA’s breakthrough designation for givosiran, formerly known as ALN-AS1. And the biotech is planning to use that inside regulatory track to hustle along a pivotal study that will launch in a matter of months.
The prominent Cambridge, MA-based biotech, which has had plenty of ups and downs over the years, says it plans to roll out some of the Phase I data it’s been gathering on givosiran in late June. Until then, the company says that regulators relied on early evidence on the “meaningful reductions in the occurrence of porphyria attacks” to provide the BTD title. Late last year the biotech noted that results” demonstrated a robust and durable lowering of aminolevulinic acid and porphobilinogen, the toxic heme intermediates that are believed to mediate porphyria symptoms and acute attacks.”
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