- Alnylam Pharmaceuticals may have to wait up to three months longer for the Food and Drug Administration to decide whether to approve its latest medicine due to issues at a third party packaging and labeling facility.
- According to Alnylam, the facility was recently inspected but still requires the FDA's signoff before it can be used to launch the drug, called vutrisiran. The company noted that none of the issues inspectors had with the facility were directly related to vutrisiran.
- In response, Alnylam has found another facility to help pack and label its medicine and has amended its approval application. The agency now expects to decide by July 14 whether to clear vutrisiran, which is designed to treat a rare and fatal disease that causes a toxic buildup of protein in various tissues, including the heart and nervous system.
A pioneer in a drugmaking technique known as RNAi, or RNA interference, Alnylam already has three products on the market. Its first, Onpattro, treats an inherited form of the rare disease transthyretin amyloidosis, and was the first ever RNAi therapy to get the FDA's stamp of approval.
Alnylam hopes to grow that list of medicines with vutrisiran. The drug is meant to be a more convenient and longer-lasting version of Onpattro, and is being used to treat patients with both inherited and non-inherited forms of transthyretin amyloidosis. Early last year, the company disclosed results from a late-stage study of patients with nerve damage from the disease, showing treatment with vutrisiran significantly reduced neurological impairment after nine months.
With those results in hand, Alnylam submitted an approval application to the FDA last April. The agency subsequently agreed to review the application and make a decision by April 14, 2022.
Now, though, a decision may not come until mid-July.
Mani Foroohar, an analyst at the investment bank SVB Leerink, wrote to clients that "the delay does take some wind out of the company's sales," and makes it less likely Alnylam will increase its financial forecast for the year. Alnylam's share price fell about 3% in Monday morning trading
Still, Foroohar expects only a "modest" impact to Alnylam from the delay, as do others.
"As we see it, because the issue related to a third-party facility was not directly related to vutrisiran, and because this facility was used for packaging and labeling ... it would seem reasonable that Alnylam, [which] has incorporated a new facility into their application, is still in a likely position to attain vutrisiran approval" by the July review deadline, Stifel analyst Paul Matteis wrote in his own note to clients.