Alnylam Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to approve Onpattro (patisiran) for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. Onpattro already is approved to treat familial amyloid polyneuropathy (FAP), a genetic disorder in which toxic tangles…
One-time treatment with the experimental gene-editing therapy NTLA-2001 led to substantial reductions in the levels of toxic transthyretin protein that were sustained for several months in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). That is according to data shared by NTLA-2001’s co-developer, Intellia Therapeutics, at the American Heart Association…
A woman with familial amyloid polyneuropathy (FAP) developed a type of kidney disease called focal segmental glomerulosclerosis — characterized by scarring in the kidneys — several months after starting treatment with Tegsedi (inotersen), according to a recent case report. Once the patient stopped taking Tegsedi, the kidney…
The European Commission has approved Amvuttra (vutrisiran) as a treatment for familial amyloid polyneuropathy (FAP) — also known as hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy — for people in the EU with stage 1 or stage 2 polyneuropathy. The decision was announced by the medication’s developer, Alnylam…
Onpattro (patisiran), a widely approved treatment for familial amyloid polyneuropathy (FAP), preserved functional ability and improved life quality for people with a related form of heart disease called transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. That’s according to top-line results from the Phase 3 APOLLO-B clinical trial, which tested the…
Alnylam Pharmaceuticals has selected Orsini Specialty Pharmacy to distribute Amvuttra (vutrisiran), which was recently approved by the U.S. Food and Drug Administration (FDA) to treat familial amyloid polyneuropathy (FAP). “We are honored to work with Alnylam in providing access to this important treatment option or medication for…
A one-time treatment with NTLA-2001, Intellia Therapeutics’ experimental gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other types of ATTR amyloidosis, continued to demonstrate reduced levels of the disease-driving transthyretin (TTR) protein for at least six months, new data show. “Based on the interim data shared today,…
Several cases in which treatment with Onpattro (patisiran) was used to ease familial amyloid polyneuropathy (FAP) symptoms were described in a recent report from Italy. The study, “Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran,” was published in Pharmacogenomics and Personalized…
Vutrisiran — now known by the brand name Amvuttra — has been approved by the U.S. Food and Drug Administration (FDA) to treat familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. The therapy is expected to be available for shipment to healthcare providers…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…