Alnylam Sees Positive Results in Latest Clinical Trials
Alnylam Pharmaceuticals has announced positive results from its latest phase 3 clinical trials of patisiran, the company’s lead drug designed to treat a rare nerve disorder known as familial amyloid polyneuropathy (FAP).
According to Stat News, the safety of patisiran in the FAP study was high quality, squashing any concerns previously raised by toxicity reports with some of Alnylam’s other pipeline drugs.
As a result of this, a European approval will follow in early 2018, much to the company’s delight after it intended to seek US marketing approval for patisiran before the end of the year.
If approved, the drug would become the first commercial product based on RNA interference, or RNAi, a technology that uses snippets of genetic code to shut down disease-causing genes. Alnylam is so nearly on the cusp of proving that RNAi can be transformed into a medicine that helps patients.
John Maraganore, Alnylam’s CEO, explained, “We are very proud to report the first ever positive Phase 3 results for an RNAi therapeutic, marking the potential arrival of an entirely new class of medicines. This moment is the culmination of a 15-year journey of tireless work by countless contributors who have overcome enormous scientific and business challenges to make RNAi therapeutics a reality.”
In the study of 225 FAP patients, which Alnylam calls APOLLO, patisiran significantly reduced a measure of nerve damage compared to placebo, achieving the primary endpoint with statisitical significance.
Alnylam said, the frequency of serious adverse events were comparable across both arms of the study and the death rate was lower for patisiran-treated patients.