AstraZeneca (AZ, NASDAQ: AZN), headquartered in the UK, announced last week that the U.S. Food and Drug Administration (FDA) has granted regulatory approval for its ligand-conjugated antisense oligonucleotide (LICA) Wainua (eplontersen) as a treatment for hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN/ATTRv-PN) in adults. Developed in collaboration with Ionis Pharmaceuticals (NASDAQ: IONS), Wainua is now the only self-administered ATTRv-PN therapy available in the U.S. via an auto-injector.
The FDA’s approval was informed by interim results from a Phase III study, which demonstrated that Wainua offers consistent and sustained improvements in serum transthyretin (TTR) concentrations, neuropathy impairment, and overall quality of life after 35 weeks of treatment.
AstraZeneca and Ionis plan to launch the orphan drug in the U.S. next month and have already submitted applications in other global markets. Additionally, Wainua is in late-stage development for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).- Flcube.com
