SMA Patient Meeting Showcases FDA Flexibility – And Appreciation From Advocates

The US FDA’s top drug and biologic review officials delivered a strong message of ongoing flexibility for new treatments of Spinal Muscular Atrophy (SMA) during an externally-led Patient Focused Drug Development meeting April 18.

The meeting, organized by the patient-advocacy group cureSMA, featured an extensive line-up of FDA participants, including Division of Neurology Products Director Billy Dunn, Office of Tissues and Advanced Therapies Director Wilson Bryan, and Office of New Drugs Rare Disease Staff Associate Director Jonathan Goldsmith. The tone and content of their remarks to the SMA community strongly suggest that the approval of Biogen Inc./Ionis Pharmaceuticals Inc