Several cases in which treatment with Onpattro (patisiran) was used to ease familial amyloid polyneuropathy (FAP) symptoms were described in a recent report from Italy. The study, “Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran,” was published in Pharmacogenomics and Personalized…

Switching to Onpattro (patisiran) following liver transplant and treatment with tafamidis lessened persistent disease progression in a patient with severe familial amyloid polyneuropathy (FAP), a case study reports. The man carried a rare mutation in the TTR gene — the most common cause of FAP — therefore, further studies…

Vutrisiran, an experimental therapy for familial amyloid polyneuropathy (FAP), continued to safely alleviate neurologic impairments and overall disability over 18 months of treatment, according to new data from the ongoing Phase 3 HELIOS-A trial. Those being given the therapy, administered as an under-the-skin injection once every three months,…

A man with familial amyloid polyneuropathy (FAP) developed eye problems years after he was found to have an uncommon disease-causing mutation called ATTRD18E, according to a case report from Canada. Abnormal findings included vitreous opacities, or eye floaters, and retinal angiopathy — a condition in which blood vessels at…

Onpattro (patisiran) is now reimbursed for patients with familial amyloid polyneuropathy (FAP) living across most regions of Canada, according to Alnylam Pharmaceuticals, the therapy’s developer. The announcement comes following a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut…

The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation RNA interference (RNAi) therapy candidate for familial amyloid polyneuropathy (FAP). Hereditary ATTR (hATTR) amyloidosis, which includes FAP, “is a rare, rapidly progressive, debilitating and fatal condition and we are…

Treatment with disease-modifying therapies can extend survival in people with familial amyloid polyneuropathy (FAP), a new study indicates. The study, “Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area,” was published in the journal Brain…

Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is being reviewed by the U.S. Food and Drug Administration (FDA), with a decision expected by April 14 of next year. The agency has indicated that it is not currently planning…

Enrollment is now completed in the Phase 3 APOLLO-B trial, which is assessing the safety and efficacy of Onpattro (patisiran) to treat heart disease in people with transthyretin-mediated (ATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The study (NCT03997383) has enrolled more than…

Editor’s note: The FAP News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read more stories from the conference. Treatment with Onpattro (patisiran) lessened signs of nervous system damage in people with familial amyloid polyneuropathy (FAP), new clinical…