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…
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…
Matrix metalloprotease-14 (MMP-14) is a novel biomarker for therapy follow-up, as well as a potential therapeutic target in familial amyloidotic polyneuropathy (FAP), said researchers in Portugal. Their study, “MMP-14 overexpression correlates with the neurodegenerative process in familial amyloidotic polyneuropathy,” appeared in the journal Disease Models and Mechanisms. FAP…
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…
The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…
Pfizer’s Vyndaqel (tafamidis) can significantly delay the progression of familial amyloid polyneuropathy, regardless of its severity before treatment or which mutation causes it, a study reports. The research, based on the combined results of two clinical trials, appeared in the European Journal of Neurology. The title of the article is “…
Alnylam Pharmaceuticals’ lead investigational therapeutic patisiran, for the treatment of hereditary ATTR (hATTR) amyloidosis, also known as familial amyloid polyneuropathy (FAP), was granted accelerated assessment by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). “We are pleased the CHMP has granted accelerated assessment for patisiran…