AstraZeneca (AZ) has received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for the use of Wainzua (eplontersen) in adults with the rare progressive condition hereditary transthyretin amyloidosis (ATTRv).
The drug has been specifically authorised for use in patients with polyneuropathy (PN), which is damage to multiple nerves outside of the brain and central nervous system. This results in pain, progressive weakness and loss of sensation in the legs and arms, as well as mobility difficulties.
In both hereditary and non-hereditary forms of transthyretin amyloidosis, transthyretin (TTR) protein builds up as fibrils in tissues and interferes with their normal functions. As the TTR protein fibrils accumulate, more tissue damage occurs and the disease worsens.
Administered as an injection under the skin using a pre-filled pen, Wainzua is designed to lower the production of TTR protein at its source, reducing the effects of the disease.
The MHRA’s decision on the therapy was supported by positive results from the late-stage NEURO-TTRansform trial, in which Wainzua-treated ATTRv patients experienced greater reductions in TTR levels and less worsening of the disease from baseline compared to an external placebo group.
Julian Beach, MHRA interim executive director of healthcare quality and access, said: “We’re assured that the appropriate regulatory standards of safety, quality and effectiveness for the approval of this new formulation have been met. As with all products, we will keep its safety under close review.”
AZ gained access to Wainua in 2021 after signing a global development and commercialisation agreement with Ionis Pharmaceuticals. The agreement outlined that the companies will jointly develop and commercialise the therapy in the US, while AZ will develop and commercialise it in the rest of the world, except in Latin America.
The partners received US Food and Drug Administration approval for Wainua to treat ATTRv-PN in December last year.
Ruud Dobber, executive vice president, biopharmaceuticals business unit, AZ, said at the time of the announcement: “There is an urgent medical need for new therapies for people living with [ATTRv-PN]. The US approval of Wainua offers a new treatment option that provides consistent and sustained reduction in serum TTR concentration compared to baseline while halting disease progression and improving quality of life for people living with this debilitating condition.”
