Orphan drug battle brews

Posted 22 October 2024 PM

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.

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