UK pharmaceutical juggernaut AstraZeneca (AZ, NASDAQ: AZN) has announced that Wainzua (eplontersen), an RNA-targeted therapy co-developed with US-based Ionis Pharmaceuticals (NASDAQ: IONS), has received a positive recommendation for approval from the Committee for Medicinal Products for Human Use (CHMP) within the European Union (EU). The treatment is intended for adults with hereditary transthyretin-mediated amyloidosis in stage 1 or stage 2 polyneuropathy, known as hATTR-PN or ATTRv-PN.
ATTRv-PN arises from mutations in the transthyroxin (TTR) protein, leading to a buildup of abnormal TTR in tissues and resultant nerve damage. This progressive disease typically results in motor disability within five years of diagnosis and is often fatal within a decade without intervention. Wainzua, an antisense oligonucleotide-Galnac conjugate, binds to TTR mRNA, prompting its degradation, which in turn suppresses the production of aberrant TTR protein and diminishes its accumulation in tissues. It was initially approved in the US, marking it as the first self-administered drug for ATTRv-PN, with monthly auto-injector use.
The CHMP’s recommendation follows the promising results of the Phase III NEURO-TTRansform study, which demonstrated that over 66 weeks, patients on Wainzua showed significant and sustained improvements in serum transthyretin (TTR) concentration and neuropathy impairment as measured by the modified Neuropathy Impairment Score +7 (mNIS+7). There were also enhancements in quality of life (QoL) as assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) compared to the external placebo control group.- Flcube.com
