Injection therapy Wainua offers ‘ray of hope’ for hATTR-PN patients
Review study suggests treatment improves quality of life
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The injection therapy Wainua (eplontersen) “offers a vital ray of hope” for people living with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), an international team of scientists wrote in a review paper.
Available data suggest that Wainua, the most recently approved hATTR-PN therapy, “keeps patients with [hATTR-PN] in good health and offers them a better quality of life,” the scientists wrote.
They noted, however, that available evidence on the therapy’s safety and efficacy is largely limited to the clinical trials that served as the basis for approval, highlighting a need for future studies to learn more about the drug. In particular, they stressed a need for more data on Wainua in people with underlying liver or kidney damage and those who are pregnant or breastfeeding.
The study, “Therapeutic Efficacy and Safety Profile of Eplontersen in Hereditary Transthyretin-Mediated Amyloidosis: A Systematic Review,” was published in Health Science Reports.
hATTR-PN is a genetic disorder in which a faulty transthyretin protein forms toxic clumps that mostly accumulate in and damage nerves outside the brain and spinal cord. This can lead to neurological symptoms, including abnormal sensations and muscle weakness. Wainua, given monthly via under-the-skin injections that patients can administer themselves, works by reducing the production of transthyretin protein. It was approved in the U.S. in 2023 to treat hATTR-PN, and has received similar authorizations in Canada, the European Union, and the U.K. (where it is sold as Wainzua).
What’s known, and what needs more study?
The team from India, Pakistan, China, and Sudan reviewed the available scientific literature on Wainua to provide an overview of what’s currently known about the therapy and highlight areas where more research is needed.
The review focused mainly on data from the Phase 3 NEURO-TTRansform clinical trial (NCT04136184), which served as the basis for Wainua’s regulatory approvals. In that trial, more than 100 people with hATTR-PN received Wainua monthly, and results were compared against findings from hATTR-PN patients given a placebo in an earlier Phase 2/3 trial (NCT01737398).
That earlier trial formed the basis for approval of Tegsedi (inotersen), an older hATTR-PN treatment that works through the same mechanism but requires more frequent dosing. Tegsedi has been discontinued in the U.S. and Canada due to low utilization.
Results from NEURO-TTRansform showed that Wainua significantly reduced blood levels of transthyretin protein and slowed the progression of neurological symptoms compared with placebo.
Participants who received the therapy also generally reported improvements in measures of life quality, whereas the external placebo group usually reported worsening life quality over time.
“Injections of [Wainua] resulted in a significant decrease in [blood transthyretin levels], improved neurological function, and better quality of life,” the researchers wrote.
The most common adverse events reported in NEURO-TTRansform included reduced vitamin A levels in the blood (which can affect eye health), vomiting, proteinuria (high protein levels in urine, which can indicate kidney damage), and vision problems.
Injection site reactions were also reported in some patients. “Patients should watch out for local injection site reactions [and] rotating injection sites is recommended to minimize localized adverse events,” the researchers wrote.
Although available evidence suggests that Wainua is generally safe and effective for treating hATTR-PN, the researchers noted several areas where data are lacking. In particular, there is not much data on Wainua’s use in people with kidney or liver impairment, and the therapy has not been studied in patients who are pregnant or breastfeeding, they said.
“It is critical to emphasize the need for more research, especially [appropriately-controlled] trials, to determine the efficacy of this medicine” in those groups, the team wrote.
The scientists also said more research is needed to determine whether Wainua is also effective for treating transthyretin amyloid cardiomyopathy (ATTR-CM), a related disease in which toxic transthyretin protein clumps mainly damage the heart.
“One of the areas of future research is the examination of the long-term efficacy and safety of [Wainua], analysis of its effectiveness in cases with manifestations of [nerve and heart damage], and patients’ initiation of treatment at the early stages of the disease,” the researchers wrote.
They noted the need for studies evaluating the effects of combining Wainua with hATTR-PN-approved therapies with different mechanisms of action.
“Although data gaps remain, [Wainua] represents a promising advancement in the treatment of [hATTR-PN],” the team concluded.
Wainua was developed by Ionis Pharmaceuticals in collaboration with AstraZeneca. Neither company was involved with the study.
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