A single infusion of YOLT-201, Yoltech Therapeutics’ investigational gene-editing therapy, appears to be safe and to effectively reduce blood levels of the disease-driving TTR protein in people with familial amyloid polyneuropathy (FAP). That’s according to preliminary data from six FAP patients given the therapy in the initial,…

A committee of the U.S. Food and Drug Administration (FDA) has endorsed Onpattro (patisiran) for the treatment of the ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage. Earlier this year, Alnylam Pharmaceuticals, which is developing Onpattro, submitted a supplemental new drug application (sNDA) to the federal agency…

Treatment with Vyndaqel (tafamidis) reduces signs of heart damage for most people with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM) associated with the A97S mutation, the most common cause of the disease in Taiwan, a study in that country shows. Notably, improvements in heart health were most pronounced among patients…

A U.S. Food and Drug Administration (FDA) advisory committee will meet Sept. 13 to discuss Alnylam Pharmaceuticals‘ application of Onpattro (patisiran) for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM), according to the company. Data from the placebo-controlled Phase 3 APOLLO-B clinical trial (NCT03997383) will be reviewed at…

Treatment with Onpattro (patisiran) for 1.5 years leads to sustained reductions in disability progression among people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or damage to the heart. That’s according to interim data from the ongoing open-label extension (OLE) portion of the Phase 3 APOLLO-B clinical trial (NCT03997383). That…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Alnylam Pharmaceuticals seeking the approval of Onpattro (patisiran) for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. According to the company, the FDA is expected to announce a decision by Oct. 8. Alnylam submitted…

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…

The first patient has been dosed in the second part of a Phase 1 trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis. The first part of the study exposed patients to single ascending doses of…

Novo Nordisk has acquired Prothena’s clinical-stage antibody PRX004, an investigational therapy for people with hereditary transthyretin amyloidosis (ATTR), which also includes familial amyloid polyneuropathy (FAP), as well as other non-hereditary forms of the disease. While the company initially will focus on developing PRX004 for the treatment of ATTR…