Drug Development

Alnylam shoots for a speedy FDA OK after their next shot at fielding a pricey rare disease drug hits PhIII goal

Alnylam $ALNY is going for it.

After racking up a positive set of data for an interim review of their pivotal study for givosiran, execs at the RNAi pioneer say they’ll hustle ahead with their second shot at a new drug approval for a rare disease, just weeks after gaining a historic green light for their lead drug patisiran.

Akshay Vaishnaw.

We’re not getting any hard data in the statement out Thursday morning, just confirmation that researchers hit their marks on statistical significance in an interim look at a key biomarker. And that leaves them on track to gain a potential accelerated approval, especially as new management at the FDA is quicker than ever in pushing out new drugs for rare diseases and cancer ahead of final efficacy and safety data.

We saw early-stage data on this drug earlier in the year, when Alnylam tracked a 75% drop in the rate of acute hepatic porphyria attacks, doing even better with a 90% drop in an extension study relative to the run-in baseline rate ahead of dosing, encouraging them to see a rising rate of efficacy with longterm dosing. There was also a big drop in levels of aminolevulinic acid, believed to be the primary disease trigger for the ultra-rare disease.

Full top line results are due in early 2019, with an application coming in ahead during Q4.

Alnylam spent years in the clinic advancing leading RNAi drugs through a series of ups and downs. But now that it’s begun to roll out new drugs, the biotech has big plans to keep the drug applications coming. These won’t be cheap, with ultra-rare conditions often sold for substantial six-figure sums.

“With these interim results in hand, we plan to meet with the FDA to discuss the results and the overall benefit-risk profile for a potential NDA submission at or around year-end in support of an Accelerated Approval. In the meantime, with enrollment in ENVISION completed ahead of schedule, we look forward to reporting topline results for the full study early next year,” says Alnylam R&D chief Akshay Vaishnaw. “If clinical efficacy and acceptable safety are confirmed in the full study, we believe givosiran has the potential to transform the lives of patients living with an AHP.”


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Scientist, Quality Control
Molecular Templates Austin, TX
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Molecular Templates Austin, TX
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Flagship Pioneering Cambridge, MA

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