Japanese experts have developed guidelines for treating people in their country with transthyretin familial amyloid polyneuropathy that are different from other countries’ guidelines. That’s because TTR-FAP manifests itself differently in Japanese than in people elsewhere. It has a broader range of symptoms in Japanese patients, for example. The guidelines are…
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…
The artificial induction of joint ossification, or surgical arthrodesis, is a safe and effective option to treat patients with rare knee structural deformities due to Charcot neuroarthropathy secondary to familial amyloid polyneuropathy (FAP), according to a case report. FAP represents a heterogeneous group of hereditary diseases caused by…
A combination of three tests that measure upper limb nerve damage and sensitivity to stimuli could help doctors distinguish between transthyretin familial amyloid polyneuropathy (TTR-FAP) and similar conditions, a study reports. Because TTR-FAP treatments are most effective when done early, the tests can improve patients’ chance of a better outcome, the…
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…
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…