Ionis grants Recordati rights to zilganersen for Alexander disease

Ionis will retain the responsibility for commercialising the RNA-targeted medicine within the US and will remain the global lead for development activities.

Under the agreement, Recordati will handle regulatory filings and oversee commercial operations outside the US, including supporting early access initiatives in line with local regulations.

Ionis will receive a $30m upfront payment as part of the deal and stands to gain further milestone payments and tiered royalties of up to the mid-20% range on annual net sales.

The investigational medicine, zilganersen, is under review by the US Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) action date set for 22 September.

Ionis CEO Brett Monia said: “Recordati combines proven rare disease and global commercialisation expertise with deep experience navigating regional and local regulatory environments and the infrastructure needed to reach patients outside the US, making them the right partner to deliver zilganersen to patients with urgency. They recognise, as we do, the high unmet need for this rare, serious and often fatal neurodegenerative disease.”

Recent clinical data announced by Ionis indicated that zilganersen achieved the primary endpoint in patients aged five years and above in a pivotal study.

Zilganersen was generally well tolerated in the study, with most adverse events reported as mild or moderate, and fewer serious treatment-emergent adverse events observed in active treatment groups compared to pooled controls.

The medicine has received breakthrough therapy, orphan drug, and rare paediatric disease designations from the FDA, as well as an orphan drug designation from the European Medicines Agency.

In September 2025, Ionis Pharmaceuticals and Sobi announced the approval of Tryngolza (olezarsen) in the EU for adults with familial chylomicronemia syndrome.

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