Alnylam's Challenge: Scaling Up And Moving Beyond The Liver

By Ben Comer, Chief Editor, Life Science Leader

On June 13, a day before Alnylam Pharmaceuticals’ 20-year anniversary as a biotech company exclusively focused on the science of RNA interference (RNAi), the FDA approved Amvuttra (vutrisiran), the company’s fifth internally discovered RNAi therapeutic to reach the market in as many years. The approval was significant for several reasons. First, Amvuttra provides a more convenient treatment option — a subcutaneous injection once every three months, compared with an 80-minute infusion every three weeks — for patients with polyneuropathy caused by hereditary transthyretin-mediated (hATTR) amyloidosis, a rare and deadly disease. Second, the approval marks an important first step in the life cycle of Amvuttra, which is being tested in a separate clinical program for ATTR patients with cardiomyopathy, a substantially larger patient population. Third, the approval is the company’s first under CEO Yvonne Greenstreet, although Greenstreet has worked for Alnylam in roles of increasing responsibility since 2016.