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.”
The FDA developed the breakthrough drug program with an eye to speed development of new and important drugs. Recently new FDA commissioner Scott Gottlieb has been advocating that all units inside the FDA emulate the success of the oncology group in making faster, more efficient clinical trials a reality. That could be key to Alnylam, which is angling for a series of late-stage programs to take the company into commercialization after 15 years of development work.
In this particular case, Alnylam execs are planning a Phase III launch later this year. Its most advanced drug is patisiran, putting givosiran into a second tier of drugs in the pipeline.
Alnylam, though, has had to deal with some alarming safety issues along the way. That may focus more attention on its note that there was one death from acute pancreatitis in the third cohort of the Phase I study, which remained blinded, so investigators don’t know if the patient was in the drug or placebo arm. Either way, the biotech says it was unlikely that the death was drug-related.
Jeff Miller, who runs the givosiran program at Alnylam, had this to say:
We believe givosiran could become a transformative treatment for patients with this devastating and potentially life-threatening disease. Accordingly, we look forward to rapidly advancing this program in collaboration with global regulatory authorities, having also received PRIME designation from the European Medicines Agency earlier this year. We plan to initiate the Phase III clinical program with givosiran in late 2017.
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