Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending ALN-TTRsc for designation as an orphan medicinal product for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR).
“We are very pleased to have received a positive opinion from the EMA COMP on our application for Orphan Drug Designation for ALN-TTRsc,” said Saraswathy (Sara) Nochur, Ph.D., Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. “We believe RNAi therapeutics represent a promising new approach for the treatment of ATTR, with the potential to make a meaningful impact for patients with this progressive and debilitating disease. We look forward to sharing Phase 2 clinical data from our ALN-TTRsc program later in the year, and, assuming continued positive results, we plan to advance to a Phase 3 pivotal trial in ATTR patients with TTR cardiac amyloidosis by the end of the year.”
ALN-TTRsc is currently in a pilot Phase 2 clinical trial for the treatment of ATTR patients with TTR cardiac amyloidosis; this study is aimed at evaluating the tolerability of ALN-TTRsc in approximately 15 patients. In addition, the study will assess preliminary clinical activity as measured by knockdown of serum TTR levels and additional exploratory tests, such as cardiac imaging (including echocardiography and cardiac MRI), circulating cardiac biomarkers (NT-proBNP and troponins T and I), 6-minute walk test, New York Heart Association (NYHA) classification, and measures of heart failure symptoms and quality of life (Kansas City Cardiomyopathy Questionnaire and EQ-5D QOL). The company expects to present data from the Phase 2 trial in late 2014. Patients completing the Phase 2 trial will be eligible to participate in an open-label extension (OLE) study for further assessment of general tolerability and clinical activity with long-term dosing; the ALN-TTRsc Phase 2 OLE study is expected to be initiated in mid-2014. Assuming positive results, Alnylam expects to begin a Phase 3 trial in TTR cardiac amyloidosis patients by the end of 2014.