December 23, 2016
FDA announced that it had provided an accelerated approval for Spinraza (nusinersen), the new drug from Biogen and Ionis, for treating rare cases of spinal muscular atrophy. It is estimated that the total sales of this drug would reach $3 billion by 2026. On this news, shares of Ionis climbed 10% and Biogen saw its stock shot up 2%. After a year of upheavals, both companies badly needed this win.
Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said: “There has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life. As shown by our suggestion to the sponsor to analyze the results of the study earlier than planned, the FDA is committed to assisting with the development and approval of safe and effective drugs for rare diseases and we worked hard to review this application quickly; we could not be more pleased to have the first approved treatment for this debilitating disease.”