The Ministery of Food and Drug Safety said on the 13th that it approved an autoinjector formulation of Wainua (ingredient name eplontersen sodium), a treatment for the rare hereditary neurologic disease transthyretin (TTR) familial amyloid polyneuropathy.
Wainua is an RNA-based antisense oligonucleotide (ASO) therapy co-developed by Britain's AstraZeneca and U.S.-based Ionis Pharmaceuticals. It was approved by the U.S. Food and Drug Administration (FDA) in 2023 and is currently sold in about 20 countries, including the United States, as a treatment for hereditary transthyretin amyloid polyneuropathy (ATTR-PN).
The newly approved product is a 45 mg autoinjector formulation that improves dosing convenience for patients. Once a month, patients place the device on the skin and press a button to automatically deliver the drug subcutaneously.
Transthyretin familial amyloid polyneuropathy is a rare disease in which genetic mutations in the TTR protein produced by the liver cause abnormal amyloid to accumulate in peripheral nerves, leading to sensory disturbances and decreased muscle strength.
The active ingredient, eplontersen sodium, is an ASO therapy that binds to the messenger RNA (mRNA) of the specific gene that causes the disease and suppresses production of the TTR protein. This reduces abnormal amyloid deposition and slows disease progression.
The approval covers patients with stage 1 or stage 2 polyneuropathy.
The Ministery of Food and Drug Safety noted that it designated the product as No. 58 under the Global Innovative product Fast-track review Support system (GIFT) and supported expedited review from the development stage.
The Ministery of Food and Drug Safety said, "We will continue to promptly review and approve innovative therapies with confirmed safety and efficacy based on regulatory science expertise to expand patients' treatment opportunities."