AstraZeneca (AZ) and Ionis’ Wainzua (eplontersen) has been recommended by the European Medicines Agency’s human medicines committee for use in adults with the rare disease hereditary transthyretin-mediated amyloidosis (ATTRv).
The Committee for Medicinal Products for Human Use (CHMP) has specifically recommended that the drug be used to treat patients with stage one or two polyneuropathy (PN), a condition that affects multiple nerves in the body and causes pain, progressive weakness and loss of sensation in the legs and arms, as well as mobility difficulties. Up to 40,000 people worldwide are living with ATTRv-PN.
Both hereditary and non-hereditary forms of transthyretin (TTR) amyloidosis occur when 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.
Wainzua, which can be self-administered monthly via an auto-injector, is designed to lower the production of TTR protein at its source in the liver.
The CHMP’s decision on the therapy was based on positive results from the late-stage NEURO-TTRansform trial, in which Wainzua-treated patients demonstrated consistent and sustained benefit on the co-primary endpoints of serum TTR concentration and neuropathy impairment compared to an external placebo group.
Wainzua was also associated with improvements in the key secondary endpoint of quality of life, and the drug continued to show a favourable safety and tolerability profile throughout the trial.
The European Commission will now consider the CHMP’s recommendation as it makes a final decision on Wainzua in this indication.
Ruud Dobber, executive vice-president, biopharmaceuticals business unit, AZ, said: “Due to the progressive nature of ATTRv-PN, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease.
“[This] 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 quality of life.”
The positive opinion comes just days after the Medicines and Healthcare products Regulatory Agency approved Wainzua to treat ATTRv-PN. The US Food and Drug Administration also authorised the drug under the brand name Wainua for the same patient population in December last year.
AZ and Ionis are commercialising Wainua for ATTRv-PN in the US, while AZ will develop and commercialise it in the rest of the world, except in Latin America.
