Alnylam says an inspection at a secondary packaging facility run by a third-party has pushed the FDA’s approval date for vutrisiran back three months.
Alnylam Pharmaceuticals is a pioneer in the RNAi space, becoming the first company to bring a small interfering ribonucleic acid (siRNA) treatment to the market with Onpattro (patisiran), approved by the US Food and Drug Administration (FDA) in August 2018.
Since then, the firm has achieved success with Givlaari (givosiran) and Oxlumo (lumasiran) but has suffered a minor setback with its investigational therapy Vutrisiran this week.
The investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, originally had an action date under the Prescription Drug User Fee Act (PDUFA) of April 14, 2022. However, issues at a contract development and manufacturing organization (CDMO) has driven the FDA to set a new goal date for July, Alnylam announced earlier this week.
“Alnylam recently learned that the original third-party secondary packaging and labeling facility the Company planned to use for the vutrisiran launch was recently inspected and the inspection requires classification for the FDA to take action on the vutrisiran NDA,” the company said in a statement.
“The inspection observations were not directly related to vutrisiran. In order to minimize delays to approval, Alnylam has identified a new facility to pack and label vutrisiran and submitted an amendment to the NDA for review by the FDA. The updated Prescription Drug User Fee Act (PDUFA) goal date to allow for this review is July 14, 2022.”
Alnylam told us it is “not at liberty to share the details of the observations of the facility at this time because of confidentiality obligations we have to the third party” when contacted for further information.