The first adult with familial amyloid polyneuropathy (FAP) has been dosed in a Phase 3 clinical trial testing nexiguran ziclumeran (nex-z), Intellia Therapeutics’ one-time gene therapy. The trial, called MAGNITUDE-2 (NCT06672237), is recruiting up to 50 adults diagnosed with FAP, also known as hereditary transthyretin (ATTR) amyloidosis…

Alnylam Pharmaceuticals is planning to launch a Phase 3 clinical trial toward the end of this year to test its therapy candidate nucresiran in people with familial amyloid polyneuropathy (FAP) — though details on the trial’s design are still being discussed with global regulators. That’s according to pipeline…

Treatment with Amvuttra (vutrisiran) may ease heart damage in people with familial amyloid polyneuropathy (FAP), a new analysis of the HELIOS-A Phase 3 clinical trial suggests. “The totality of assessments in this exploratory analysis of the HELIOS-A study provides evidence of potential benefit of [Amvuttra] on cardiac [heart-related] manifestations…

Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study, which is expected to start by the end of the year, follows the recent…

Alnylam Pharmaceuticals announced that it has completed patient enrollment for its Phase 3 HELIOS-A trial of vutrisiran, which will test the safety and efficacy of the candidate therapy for treating hereditary transthyretin amyloidosis. HELIOS-A (NCT03759379) enrolled 160 participants from 68 clinical sites…

A Phase 3 trial testing patisiran as a therapy for hereditary ATTR amyloidosis with polyneuropathy (also called familial amyloid polyneuropathy, or FAP), showed treatment with patisiran safely improved many symptoms as well as patients’ quality of life. The full results have been published in The New England…

Akcea Therapeutics has completed a deal giving it the global marketing rights to its affiliate  Ionis Pharmaceuticals’ familial amyloid polyneuropathy therapy inotersen. The deal also gives Akcea rights to a follow-up treatment known as AKCEA-TTR-L Rx, formerly known as IONIS-TTR-L Rx. Inotersen was designed for hereditary forms of FAP, which…

Ionis Pharmaceuticals has asked the European Medicines Agency (EMA) to approve its lead drug candidate inotersen to treat hereditary TTR amyloidosis (ATTR), also known as familial amyloid polyneuropathy (FAP). The request, known as a Marketing Authorization Application (MAA), is supported by positive data from the Phase 3 NEURO-TRR trial, and…

The latest results of the Phase 3 NEURO-TTR study continue to demonstrate the therapeutic potential of inotersen for patients with hereditary TTR amyloidosis (hATTR) with polyneuropathy, also known as familial amyloid polyneuropathy (FAP). The investigational drug developed by Ionis Pharmaceuticals showed clinically meaningful benefits and improved quality of…

An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the investigational treatment delays disease progression, Pfizer announced. The study, “Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid…