A Global Step Forward for Zilganersen
A few months out from a pivotal moment in the U.S., there's encouraging news for the Alexander disease community around the world.
On June 25, 2026, Ionis Pharmaceuticals announced a licensing agreement with Recordati, a global rare disease company. Under the agreement, Recordati gains exclusive rights to develop and commercialize zilganersen — Ionis's investigational RNA-targeted medicine for Alexander disease — in all countries outside the United States. Ionis will keep sole commercial responsibility for zilganersen in the U.S. and will continue to lead its development globally.
Why this matters to families outside the U.S.
Bringing a rare disease treatment to patients isn't just about the science. It's about navigating each country's regulatory pathways, access programs, and approvals — a process that looks different everywhere. Recordati brings deep rare disease experience and the on-the-ground infrastructure to do exactly that. In plain terms: there's now a committed partner whose job is to help bring zilganersen to AxD families beyond the United States.
Where things stand in the U.S.
Zilganersen is currently under FDA review, with a decision (PDUFA) date of September 22, 2026. If approved, it would be the first disease-modifying treatment for Alexander disease — and Ionis's first independent commercial launch in neurology. This follows recently reported positive results from the pivotal study, where zilganersen met its primary endpoint by showing meaningful stabilization of walking ability in participants age five and older, alongside a favorable safety profile.
An honest word
Zilganersen is still investigational. It is not yet approved in the United States or anywhere else, and approval is never guaranteed. We share this news not to overstate hope, but because it's a genuine, concrete step toward the day this treatment can reach the people who need it — wherever they live.
We'll continue to follow this closely and keep our community informed every step of the way.
Read the full announcement from Ionis here:
