Author Archives: Magdalena Kegel

While Vyndaqel (tafamidis) has been shown to improve symptoms of familial amyloid polyneuropathy (FAP), it likely does not protect against anemia — a common problem among patients with the disease, a small Japanese study found. The data suggests that physicians may need to pay attention not only to patients’ neurological…

Alnylam Pharmaceuticals and Sanofi have dissolved the partnership they formed to develop and market two treatments for the neurological disease familial amyloid polyneuropathy, or FAP. The change is aimed at streamlining the development of patisiran and ALN-TTRsc02, Alnylam said in a press release. The two drugs are RNAi treatments. This means…

The U.S. Food and Drug Administration (FDA) has agreed to speedily review Ionis Pharmaceuticals’ application to get  its investigational drug inotersen approved as a treatment for hereditary TTR amyloidosis (hATTR). The application has been granted Priority Review status, shortening the review process from 10 to six months. The FDA…

An assessment of the global prevalence of familial amyloid polyneuropathy, caused by mutations in the transthyretin (TTR) gene, gave a similar count to that estimated in earlier studies. But the uppermost number in its range — that of 38,468 affected people — was notably higher. That upper estimate, in the study funded…

Alnylam Pharmaceuticals and its partner Sanofi Genzyme have submitted an application that asks European regulators to approve patisiran for adults with hereditary transthyretin-mediated (hATTR) amyloidosis. The European Medicines Agency (EMA) announced earlier that it will review the so-called Marketing Authorisation Application (MAA) under an accelerated assessment pathway, which will…

Alnylam Pharmaceuticals has submitted a complete new drug application (NDA) for its investigational therapy patisiran to the U.S. Food and Drug Administration for the treatment of adults with hereditary ATTR amyloidosis, also called familial amyloid polyneuropathy (FAP). The application has been submitted on a rolling basis — a…

Researchers have found that a large proportion of untreated people with the TTRVal30Met mutation — causing familial amyloid polyneuropathy (FAP) — have cognitive impairment, particularly if they experience disease symptoms. Such problems are more common in older patients and those with late-onset disease, the study, published in the Journal of Neurology…

Alnylam Pharmaceuticals has started its submission of a rolling new drug application (NDA), asking the U.S. Food and Drug Administration (FDA) to approve patisiran for the treatment of hereditary ATTR amyloidosis, also called familial amyloid polyneuropathy (FAP). The rolling submission allows the company to submit pieces of the required…

Alnylam Pharmaceuticals, together with its partner Sanofi Genzyme, has now released the full results from the Phase 3 trial of patisiran — a drug intended to treat hereditary ATTR amyloidosis with polyneuropathy, also called familial amyloid polyneuropathy (FAP). The study’s findings — presented at last week’s First European…

Patients with familial amyloid polyneuropathy (FAP) appear to have intact reproductive capacity, even when they have undergone a liver transplant or receive treatment with Vyndaqel (tafamidis), a retrospective study suggests. Researchers from the University of Porto, in Portugal, reached this conclusion after studying FAP patients carrying the V30M mutation…