Ionis Pharma Says FDA Grants Fast Track Designation For Zilganersen To Treat Alexander Disease

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) announced Tuesday that the U.S. Food and Drug Administration (FDA) has granted zilganersen Fast Track designation for the treatment of children and adults with an ultra-rare, progressive and ultimately fatal neurological disorder known as Alexander disease (AxD).

Zilganersen is the first investigational medicine in clinical development for adults and children living with Alexander disease.

The pivotal Phase 1-3 study of zilganersen in adults and children living with AxD completed enrollment earlier this year across 13 sites in eight countries.

The topline data from the pivotal study of zilganersen is expected in the second half of 2025.

The FDA grants investigational medicines Fast Track designation to facilitate the development and expedite the review of medicines that demonstrate the potential to treat serious conditions and fill an unmet medical need.