Patricia Inácio, PhD, science writer —

The U.S. Food and Drug Administration has agreed to Alnylam Pharmaceuticals’ request to expand the orphan drug designation for patisiran to include a second type of familial amyloidosis. Patisiran’s original orphan designation was for familial amyloidotic polyneuropathy, the most common form of the disease. The new designation covers a form…

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…

A new clinical trial called TRAM aims to determine the prevalence of genetic mutations in the transthyretin (Ttr) gene, the underlying cause of TTR familial amyloid polyneuropathy (TTR-FAP). The trial (NCT03237494) is for adult patients with polyneuropathy disease or cardiomyopathy of unknown causes and is currently recruiting participants. Mutations…

In the largest registry worldwide for a common subtype of familial amyloid polyneuropathy, known as FAP ATTRV30M, researchers identified six genes variants that influence the age at which disease symptoms become evident in a person and that may point the way to new treatments. The study, “Familial amyloid polyneuropathy…