Five years of treatment with Onpattro (patisiran) slowed disability progression and maintained quality of life for people with familial amyloid polyneuropathy (FAP) in a long-term clinical trial, a study shows. Although Onpattro can help delay FAP progression, “patients tend not to recover function that is lost before starting…

Onpattro (patisiran) may help familial amyloid polyneuropathy (FAP) patients who respond poorly to tafamidis meglumine stabilize or have reduced symptoms of nerve damage, called polyneuropathy. The findings are part of of a real-world study in France that analyzed data from 24 patients on tafamidis meglumine, approved as…

A U.S. Food and Drug Administration (FDA) advisory committee will meet Sept. 13 to discuss Alnylam Pharmaceuticals‘ application of Onpattro (patisiran) for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM), according to the company. Data from the placebo-controlled Phase 3 APOLLO-B clinical trial (NCT03997383) will be reviewed at…

Onpattro (patisiran), a liver-directed gene-silencing therapy, successfully slowed progression of symptoms that emerged in two brothers with hereditary transthyretin amyloidosis (hATTR) decades after their disease was stabilized by liver transplants, a study showed. In the reemergence of disease symptoms, the brothers showed also brain and eye problems that were…

Three recently approved therapies for treating familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin amyloidosis with polyneuropathy, have won their developers Best Biotechnology Product honors in the 2020 Prix Galien USA Awards, the Galien Foundation announced. Ionis Pharmaceuticals and its subsidiary Akcea Therapeutics claimed the…

Tegsedi (inotersen) and Onpattro (patisiran) are both effective treatments to stop the progression of familial amyloid polyneuropathy (FAP), alleviate gastrointestinal symptoms, and improve patients’ overall quality of life, a review study has found.  The study, “Novel RNA-targeted…

Alnylam Pharmaceuticals announced the submission of a New Drug Application (NDA) to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for  the approval of patisiran as a treatment for hereditary transthyretin-mediated (hATTR) amyloidosis. hATTR amyloidosis, also known as familial amyloid polyneuropathy (FAP), is caused by mutations in the TTR gene. It…

The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ Onpattro (patisiran) as the first-ever treatment for patients in the U.S. with familial amyloid polyneuropathy (FAP). “Today’s historic approval marks the arrival of a first-of-its kind treatment option for a rare and devastating condition with limited treatment options,”…