Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration has extended the review timeline of the New Drug Application for vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated amyloidosis, to allow for the review of newly added information related to the new secondary packaging and labeling facility. 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 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 goal date to allow for this review is July 14, 2022. No additional clinical data have been requested by the FDA.
“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. We are confident in our regulatory application and the body of data supporting the efficacy and safety of vutrisiran.”
Pushkal Garg, M.D., Chief Medical Officer & EVP, Development and Medical Affairs
Vutrisiran is also under review by the European Medicines Agency the Brazilian Health Regulatory Agency and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). We aim to minimize the impact of this issue on these reviews through timely resolution of the matter or utilization of an alternative facility for one or more of these regions. Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis, and in Japan for transthyretin type familial amyloidosis with polyneuropathy.
About Vutrisiran
Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hATTR and wild-type ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of wild-type and variant transthyretin protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization ChemistryGalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections.
About Alnylam Pharmaceuticals
Alnylam has led the translation of RNA interference into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO®, GIVLAARI® (givosiran), OXLUMO® and Leqvio® being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefitingpatients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.