AstraZeneca's Wainzua Approved by MHRA for Rare Nerve Disease ATTRv-PN

AstraZeneca (AZ) has recently secured approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for the use of Wainzua (eplontersen) in adults suffering from hereditary transthyretin amyloidosis (ATTRv), a rare and progressive condition. This drug is specifically sanctioned for patients experiencing polyneuropathy (PN), which involves damage to multiple peripheral nerves, resulting in pain, progressive weakness, and loss of sensation in the limbs, alongside difficulties in mobility.

Transthyretin amyloidosis, both hereditary and non-hereditary, is characterized by the buildup of transthyretin (TTR) protein in the form of fibrils within tissues, which hampers their normal functions. The accumulation of TTR protein fibrils leads to increasing tissue damage and worsening of the disease.

Wainzua is administered as an injection under the skin using a pre-filled pen. It is crafted to reduce the production of TTR protein at its source, thereby mitigating the effects of the disease. The MHRA's approval of this therapy was based on positive outcomes from the late-stage NEURO-TTRansform trial. In this trial, patients treated with Wainzua showed greater reductions in TTR levels and experienced less disease progression compared to an external placebo group.

Julian Beach, the interim executive director of healthcare quality and access at MHRA, expressed confidence in the regulatory standards met by this new formulation: "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."

In 2021, AZ obtained access to Wainzua through a global development and commercialization agreement with Ionis Pharmaceuticals. This agreement delineated that the two companies would co-develop and commercialize Wainzua in the United States, while AZ would handle its development and commercialization in the rest of the world, excluding Latin America. The partners received approval from the US Food and Drug Administration (FDA) for Wainzua to treat ATTRv-PN in December of last year.

Ruud Dobber, the executive vice president of AZ's biopharmaceuticals business unit, highlighted the urgency of new therapies for people with ATTRv-PN: "There is an urgent medical need for new therapies for people living with [ATTRv-PN]. The US approval of Wainzua provides a new treatment option that ensures 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."

The approval of Wainzua marks a significant advancement in the treatment of hereditary transthyretin amyloidosis, offering hope to patients with this challenging and progressive disease. The collaborative efforts of AstraZeneca and Ionis Pharmaceuticals have culminated in a therapy that addresses a critical need in the medical community, potentially transforming the lives of those affected by ATTRv-PN.