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The agency has extended the review period to address pending inspection classification at third-party secondary packaging and labeling facility. The new PDUFA date is July 14, 2022.
The FDA has extended the review timeline of Alnylam Pharmaceuticals’ new drug application (NDA) for vutrisiran, an investigational RNAi therapeutic to treat patients with transthyretin-mediated (ATTR) amyloidosis. The review has been extended three months to allow regulators to review newly added information related to the new secondary packaging and labeling facility.
No inspection observations were directly related to vutrisiran and no additional clinical data have been requested by FDA, the company said in a statement.
A third-party secondary packaging and labeling facility Alnylam 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. 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. No additional clinical data have been requested by the FDA.
Vutrisiran is administered subcutaneously every three months and treats both both hereditary ATTR (hATTR) amyloidosis and wild-type ATTR (wtATTR) amyloidosis.
“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 the polyneuropathy of hATTR amyloidosis, an inherited, progressively debilitating, and fatal disease,” Pushkal Garg, M.D., chief medical officer and executive VP, development and medical affairs, said in a press release.
ATTR amyloidosis is a protein misfolding disorder, according to the Cleveland Clinic. Transthyretin is a protein made by the liver that helps carry thyroid hormone and vitamin A in the blood. It can lead to amyloid deposits in the heart and/or the nerves and other organs and tissues.
It is a rare disease, impacting about 1 in 100,000 Americans of European descent, according to the National Library of Medicine. The cardiac form of transthyretin amyloidosis is more common among people with African ancestry. It is estimated that this form affects between 3% and 3.9% of African Americans and about 5% of people in some areas of West Africa.
The FDA had accepted the application for vutrisiran in June 2021. The submission was based on results of the phase 3 HELIOS-A study, which found that vutrisiran met the primary and all secondary endpoints, with statistically significant improvements in neuropathy, quality of life, and gait speed, and demonstrated an encouraging safety profile, relative to the external placebo group of the APOLLO study of Onpattro (patisiran), Alnylam’s therapy that is approved to treat polyneuropathy caused by hereditary ATTR (hATTR) amyloidosis.
Applications for vutrisiran are also under review by European Medicines Agency, the Brazilian Health Regulatory Agency, and the Japanese Pharmaceuticals and Medical Devices Agency.