- Alnylam Pharmaceuticals Inc. will continue to source active pharmaceutical ingredient for its experimental RNAi therapeutic patisiran from Agilent Technologies Inc., recently inking a five-year manufacturing services deal with the laboratory services provider.
- Agilent has worked with Alnylam over the past decade, producing the API of patisiran as the rare disease drug wended its ways through clinical testing. Now, as Alnylam awaits a decision on U.S. approval, the companies are paving the way for ensuring commercial supply.
- Per deal terms, disclosed in a regulatory filing April 3, Agilent is required to provide a certain but undisclosed percentage of patisiran drug product slated for commercial sale.
The Food and Drug Administration is expected to decide on approval by this August. If the agency OK's the drug, it will become the first RNA interference-based therapeutic cleared for sale in the U.S. — a milestone the biotech has been chasing for nearly two decades.
Patisiran works through a cellular process known as RNA interference, or RNAi, where the insertion of strands of short interfering ribonucleic acid can effectively "silence" genes. Rather than blocking the action of a protein, patisiran and other RNAi therapeutics disrupt the signal sent from genes through messenger RNA.
Alnylam designed patisiran to treat a rare disease known as hereditary TTR amyloidosis, caused by the build-up of TTR protein in organs.
Agilent has supplied the nucleic acid API to Alnylam since 2007 and, under the newly inked deal, will continue to do so through potential commercialization.
Alnylam has the ability to produce some bulk drug product in house, and has begun construction of a Good Manufacturing Practices-compliant facility in Norton, Massachusetts to supply drug substance in the future. That plant is set to come on line in 2020, according to Alnylam's latest filing with the Securities and Exchange Commission.
Unfortunately for RNAi drug developers like Alnylam, the interfering strands of nucleic acid aren't easily deliverable to their target.
Solving the delivery problem took Alnylam nearly a decade, leading the biotech to license a lipid nanoparticle platform from Arbutus Biopharma Corp. in 2012. Alnylam has also developed another delivery vehicle known as GalNAc, although that won't be used for patisiran.
If patisiran wins approval, it will likely face competition from Ionis Pharmaceutical Inc.'s inotersen, which is expected to secure an OK a month before in July. And recent positive results from a late-stage study of Pfizer Inc.'s tafamidis could usher in a new rival as well.