The US Food and Drug Administration (FDA) has granted Fast Track designation to vutrisiran (ALN-TTRSC02, Alnylam Pharmaceuticals, Cambridge, MA), an investigational therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. According to the FDA, Fast Track designation is designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. With this designation, Alnylam will be eligible to submit a rolling New Drug Application for vutrisiran.
“Vutrisiran has demonstrated an encouraging safety profile in the phase 1 study, with infrequent quarterly dosing with low-volume, subcutaneous administration which potentially reduces the burden of care for this progressive, life-threatening and multisystem disease. We are therefore pleased that the FDA has granted vutrisiran Fast Track designation,” said Rena Denoncourt, vutrisiran program leader at Alnylam. “After completing enrollment earlier this year, we look forward to sharing topline results of the HELIOS-A phase 3 study of vutrisiran in early 2021. More broadly, we remain committed to developing additional therapeutic options for the treatment of ATTR amyloidosis to augment the market-leading position of Onpattro (patisiran), approved for the treatment of the polyneuropathy of hATTR amyloidosis in adults.”
In addition to Fast Track designation, vutrisiran has been granted Orphan Drug designation in the US and the European Union for the treatment of ATTR amyloidosis. The safety and efficacy of vutrisiran are being evaluated in the ongoing HELIOS-A (NCT03759379) and HELIOS-B (NCT04153149) phase 3 clinical trials. Together, these studies comprise a comprehensive clinical development program intended to demonstrate the broad impact of vutrisiran across the multisystem manifestations of disease and the full spectrum of patients with ATTR amyloidosis.