See the DrugPatentWatch profile for Wainua
Wainua, an investigational therapy, is currently under review by regulatory authorities in the European Union [1]. The European Medicines Agency (EMA) is evaluating Wainua for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) [1][2].
How is Wainua being reviewed by the EMA?
The EMA is conducting a standard review of Wainua's marketing authorization application [1]. This process involves an in-depth assessment of the drug's quality, safety, and efficacy before a decision is made on its approval for use in the EU [1].
What is Wainua intended to treat?
Wainua is being developed for adults with transthyretin amyloid cardiomyopathy (ATTR-CM) [2]. This is a rare, progressive, and life-threatening disease caused by the misfolding and accumulation of transthyretin protein in organs, including the heart [2].
What is the clinical data supporting Wainua's review?
The EMA's review is based on data from the Phase 3 ATTRibute-CM study, which evaluated Wainua in patients with ATTR-CM [2]. This study demonstrated that Wainua met its primary endpoint by significantly reducing the levels of serum transthyretin [2]. The study also showed a notable reduction in a key marker of cardiac burden, N-terminal pro-B-type natriuretic peptide (NT-proBNP) [2].
When might Wainua receive a decision from the EMA?
While the EMA is reviewing Wainua, a specific timeline for a regulatory decision has not been publicly disclosed. The duration of the review process can vary depending on the complexity of the data and the agency's workload.
What is the mechanism of action for Wainua?
Wainua is a first-in-class, oral co- P-gp and BCRP transporter substrate designed to reduce the production of transthyretin (TTR) protein in the liver [2]. By lowering TTR levels, it aims to prevent the formation and deposition of amyloid fibrils in tissues [2].
Are there other drugs approved for ATTR-CM in the EU?
Yes, other therapies are available for ATTR-CM in the EU. For instance, tafamidis is approved for the treatment of wild-type or hereditary ATTR-CM in adults [3].
What are the potential risks associated with Wainua?
The provided information does not detail specific risks associated with Wainua. However, as with any investigational drug, potential side effects and safety concerns are thoroughly evaluated during the regulatory review process [1].
Who is developing Wainua?
Wainua is being developed by BridgeBio Pharma's affiliate, Pellepharm [1].
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Sources
[1] https://www.drugpatentwatch.com/news/wainua-regulatory-status-eu
[2] https://www.bridgebio.com/news-and-media/news-releases/news-release-details/bridgebio-pharma-announces-submission-of-marketing-authorization-application-for-wainua-tm-trifrrol-for-patients-with-att-amyloid-cardiomyopathy-in-europe/
[3] https://www.ema.europa.eu/en/medicines/human/EPAR/vyndaqel-vyndamax