FDA Pushes Decision on Vutrisiran to July After Packaging Site Issues

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by Marisa Wexler MS |

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The U.S. Food and Drug Administration (FDA) is extending by three months its review timeline for vutrisiran, an experimental therapy for familial amyloid polyneuropathy (FAP) developed by Alnylam Pharmaceuticals.

A decision, originally anticipated this month, is now expected by July 14.

According to Alnylam, the FDA recently inspected a third-party facility that was to be in charge of the packaging and labeling of vutrisiran, should it be approved for commercial use. Some issues were noted in the inspection, Alnylam stated without specifying. None were reported to be directly related to vutrisiran.

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To minimize delays toward a potential approval, Alnylam has selected a new facility to pack and label the therapy. The company also submitted an amendment to its original application seeking vutrisiran’s approval. The extended FDA review is to allow time for the agency to go over this new information.

The FDA did not request additional clinical data related to vutrisiran.

“We are committed to working with the FDA and the new facility to bring this important treatment option as quickly as possible to patients living with [FAP],” Pushkal Garg, MD, chief medical officer and executive vice president of development and medical affairs at Alnylam, said in a press release.

Vutrisiran is also under review for potential approval to treat FAP in Europe, Japan, and Brazil.

Alnylam’s approval applications are supported by data from the Phase 3 HELIOS-A (NCT03759379) trial. This study, which enrolled 164 adults with FAP, compared vutrisiran with Onpattro (patisiran), an approved FAP treatment also developed by Alnylam.

Both Onpattro and vutrisiran work by reducing the production of transthyretin, the faulty protein that tends to form abnormal toxic clumps throughout tissues in FAP. In HELIOS-A, participants were given either vutrisiran (25 mg) as an under-the-skin injection every three months, or Onpattro (0.3 mg/kg) as an infusion directly into the bloodstream once every three weeks.

HELIOS-A data after 18 months of treatment showed that vutrisiran eased neuropathy symptoms, lessened disability, and improved patients’ quality of life. Treatment was generally well-tolerated, with the most common treatment-associated adverse events being pain in the extremities and joint stiffness.

“We are confident in our regulatory application and the body of data supporting the efficacy and safety of vutrisiran,” Garg said.