Ionis Pharma’s Drug for Rare Disease With No Approved Therapies Meets Goals of Pivotal Study

Patients who have a particular genetic disease whose symptoms include progressively worsening muscle control were able to walk faster following treatment with an experimental Ionis Pharmaceuticals genetic medicine, meeting the main goal of a pivotal study. Based on these results, the drugmaker said it plans a regulatory submission for what could become the first FDA-approved therapy for this ultra-rare condition.

The Ionis drug, zilganersen, was developed to treat Alexander disease , an inherited disorder that leads to neurological deterioration and symptoms that include the loss of functional mobility, inability to control muscles, and difficulty breathing and swallowing. As these symptoms worsen, they eventually become fatal. Current treatment of this ultra-rare disease, which occur