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Alnylam flags looming FDA application for givosiran, adding another round of stellar efficacy data from early study

Alnylam $ALNY is buffing up its data on givosiran in anticipation of launching an assault on an accelerated approval later in the year.

The biotech is offering up some long-term extension data from a small, early-stage trial of its number two RNAi drug in the pipeline. Researchers are sticking with the low dose of the drug after tracking a big drop in the rate of acute hepatic porphyria attacks along with levels of aminolevulinic acid, believed to be the primary disease trigger for the ultra-rare disease, and the biomarker for porphobilinogen.

Akin Akinc

Following up on its first set of positive numbers for the Phase I trial, investigators tracked a 75% drop in the rate of 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.

“We view these new results with givosiran as very encouraging, since they demonstrate robust and what we believe to be clinically meaningful reductions in urinary ALA, porphyria attack rate, and hemin administration with continued dosing for up to nearly two years,” said Akin Akinc, who’s running the program at Alnylam. “We also believe our new data support use of a monthly dosing regimen for sustained reductions in ALAS1 mRNA and urinary ALA, with improved clinical activity.”

Akinc also noted in a statement that researchers are close to enrolling the 30th patient in their ongoing Phase III study, leaving them within shot of interim data that could be used to support an NDA filing before the end of this year.

If they’re proven right, that would be the second drug Alnylam has pitched to the FDA, with patisiran currently under review and tipped to muscle aside a rival therapy developed by Ionis. Givosiran jumped up in the queue at Alnylam after the biotech was forced to abandon a program for its hereditary ATTR amyloidosis med revusiran in 2016 after monitors tracked a higher rate of deaths in the drug arm. 

Alnylam’s drugs have been dogged by safety issues through its history, but appears poised to make the leap into commercialization work with patisiran possibly followed quickly by givosiran.

The Alnylam group has noted a number of adverse events among the patients treated with givosiran, adding one that they say is likely linked to their drug. A patient with a history of allergic reactions experienced an anaphylactic reaction after their third dose of givosiran and discontinued from the study.


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