Eplontersen approved as Wainzua in EU to treat adults with FAP
Decision makes therapy first, only one injected under the skin with prefilled pen
Wainzua (eplontersen) is now approved in the European Union (EU) for treating adults with early-stage familial amyloid polyneuropathy (FAP).
With the European Commission’s approval, Wainzua can treat adults in the first two stages of FAP — stage 1, when abnormal sensations appear, but the patient is still able to walk unaided, and stage 2, when the patient can walk, but needs help.
The decision makes Wainzua, which is marketed as Wainua in the U.S., the first and only FAP treatment in the EU that can be injected under the skin by patients or their carers using a prefilled pen. The medication works to reduce the production of the faulty transthyretin protein that drives nerve cell damage in FAP.
“Today’s approval of Wainzua in Europe offers adults with [FAP] a new, self-administered treatment option that provides consistent suppression of transthyretin production and improves [nerve damage] impairment and quality of life,” Brett P. Monia, PhD, the CEO of Ionis Pharmaceuticals, the therapy’s co-developer along with AstraZeneca, said in a company press release.
The medication is approved for adults with FAP in the U.S., Canada, and the U.K. Ionis is developing and marketing the therapy with AstraZeneca in the U.S., while AstraZeneca holds its exclusive marketing rights outside the U.S.
“With approvals in North America, U.K., and now across the EU, we are proud of the continued progress as we and our partner, AstraZeneca, rapidly and effectively deliver Wainzua to people around the world,” Monia said.
The companies are seeking regulatory approval in other countries, according to the release.
In FAP, a faulty form of transthyretin protein builds up as toxic clumps, called amyloids, around nerves outside the brain and spinal cord, leading to damage.
Improvements with Wainzua treatment
Wainzua is designed to deliver to liver cells, the main transthyretin producers, a short piece of genetic material that can bind to and block the RNA molecule that serves as a template to produce transthyretin from its genetic code.
Given once a month in the abdomen, upper thigh, or upper arm, the therapy should lower levels of faulty transthyretin and slow the progression of FAP-related nerve damage.
Its approval in the European Union follows the opinion of an advising committee to the European Medicines Agency that recommended Wainzua for adults with stage 1 or 2 FAP. This was based on positive data from NEURO-TTRansform (NCT04136184), a Phase 3 clinical trial that involved 144 adults with stage 1 or 2 FAP.
Wainzua was found to be more effective than an external placebo group at slowing the nerve damage caused by the disease. The external placebo group came from a trial that tested Tegsedi (inotersen), an older FAP-approved treatment also developed by Ionis.
The main measures of efficacy included changes in blood transthyretin levels, disability related to nerve damage, and quality of life. By week 66, after about 15 months of treatment, patients on Wainzua had a significantly greater reduction in transthyretin over those on the external placebo group (81.7% vs. 11.2%).
Wainzua-treated patients also saw a significantly lower increase in average modified Neuropathy Impairment Score +7 (mNIS+7), a measure of disability related to nerve damage (0.3 vs. 25.1 points). In the mNIS+7, higher scores indicate more severe disability. Also, scores of the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (QoL-DN), a measure of quality of life where higher scores indicate worse life quality, increased by an average of 5.5 points in Wainzua-treated patients and dropped by 14.2 points in the placebo group.
The NEURO-TTRansform study ran for up to 85 weeks, or more than 1.5 years, during which Wainzua continued to work as expected and remained safe and well tolerated.
After completing the trial, its participants were eligible to enter an ongoing open-label extension (NCT05071300) to receive Wainzua once a month for up to three years.
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