Approvals

Wainzua (eplotersen), produced by AstraZeneca and Ionis, has been recommended for approval in the European Union (EU) by the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP). The drug will be used as a treatment for hereditary transthyretin-mediated amyloidosis in adult patients who have stage 1 or 2 polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN).

The CHMP opinion was informed by results from the NEURO-TRransform phase 3 trial. The data showed Wainzua consistently demonstrated sustained benefit on the co-primary endpoints of neuropathy impairment and serum transthyretin (TTR) concentration over the course of 66 weeks.

The secondary endpoint measured quality of life (QoL), using the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), versus an external placebo.

“Due to the progressive nature of polyneuropathy of hereditary transthyretin-mediated amyloidosis, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease,’ said Ruud Dobber, executive vice-president, BioPharmaceuticals Business Unit, AstraZeneca. ‘Today’s recommendation brings Wainzua one step closer for patients in Europe and, if approved, will offer a new treatment option that can provide consistent TTR suppression and results in improved QoL.”

Within five years of diagnosis, ATTRv-PN leads to peripheral nerve damage with motor disability, according to the press release. Without treatment, it is generally fatal within a decade.