Orphan drug battle brews

AstraZeneca's Wainzua has passed muster at the CHMP's October meeting, signalling that the company could file the drug in Australia, which would see it go up against Medison Pharma.

The EMA's advisory committee has recommended the antisense oligonucleotide inhibitor, marketed under the brand name Wainua in the US by Ionis Pharmaceuticals, to treat stage 1 or 2 polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis, a rare and often fatal disease that affects 40,000 people globally.

AstraZeneca secured exclusive ex-US commercialisation rights to the drug from Ionis in 2021 with a US$200 million upfront payment. Under the deal, AstraZeneca manufactures the commercial supply and will pay Ionis up to US$485 million in development and approval milestones, plus US$2.9 billion in sales-relates milestones.