Over the past few months, Team Thrivewell has been proud to be the first and only infusion center in Brooklyn to administer the orphan drug Onpattro (patisiran-LNP) by Alnylam Pharmaceuticals, Inc. The first FDA approved RNA interference (RNAi) drug ever released (Aug. 2018), Onpattro (patisiran-LNP), is the only effective alternative to orthotopic liver transplantation (OLT) available in the US for patients with Transthyretin (TTR)-mediated amyloidosis (ATTR), a rare, progressive and debilitating disease, originating from mutations in the TTR gene, leading to the production and accumulation of defective amyloid proteins throughout the body, and ultimately causing intractable peripheral neuropathy and heart failure.
We are therefore pleased to announce the Alnylam team has now invited us to participate in the Post-Authorization Safety Study (PASS) trial, a prospective, multi-center, observational study to monitor and assess the long term safety of Onpattro (patisiran-LNP) in a real-world cohort of hATTR amyloidosis patients which will span over the next 10 years. Dr. Vikram Sengupta, our medical director, will serve as Primary Investigator.
In caring for these patients we have refined and tested our model of personalized and improved access to care in patients with chronic illness who have often had long, isolating struggles, circuitous paths to diagnosis, and arduous days spent commuting to distant infusion sites for treatments they must receive every three weeks, often spending more than 18 hours to receive an infusion administered over little more than an hour. We are so grateful to have had this opportunity to serve and to promote community among these patients, many of whom come with family members for their infusions on the same day.
We are excited to be partnering with Alnylam Pharmaceuticals to formally contribute to the body of academic medical knowledge surrounding the drug which will undoubtedly help refine its use in the coming years.