AstraZeneca and Ionis' amyloidosis injectable, Wainzua, has been recommended for approval by the EU's CHMP.
The therapeutic, otherwise called eplontersen, is suitable for adult patients with transthyretin-mediated amyloidosis.
Patients should also be exhibiting stage one or two polyneuropathy.
If approved, Wainzua will be the first and only approved medicine for polyneuropathy that can be self-administered.
The CHMP's positive opinion is based on the results of the Phase III NEURO-TTRansform trial, which found that the drug could reduce patient transthyretin (TTR) concentration and neuropathy impairment.
During the 66 week trial, quality of life was also improved in patients, while the drug continued to demonstrate its tolerability and safety.
What is Wainzua?
Wainzua is a monthly silencer that provides upstream supression of TTR production.
The RNA-targeted medicine targets the liver's production of TTR, and can treat all types of transthyretin-mediated amyloidosis.
Eplontersen is currently being evaluated in the CARDIO-TTRansform Phase III trial for treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), the largest of all ATTR-CM trials to date including over 1,400 participants.
Dr Laura Obici, Head of Rare Diseases Unit, Consultant at the Amyloidosis Research and Treatment Centre Istituto Di Ricovero e Cura a Carattere Scientifico Fondazione Policlinico San Matteo, Pavia, Italy, said: “This debilitating disease is ultimately fatal if left untreated and can have a significant impact on many aspects of patients’ and caregivers' day-to-day lives."
"Having additional amyloidosis treatment options designed to reduce the production of TTR protein at its source would potentially give patients more time and ability to do what matters most to them and offer the hope of living longer with a higher quality of life."
Ruud Dobber, Executive Vice-President, BioPharmaceuticals Business Unit, AstraZeneca, said: “Due to the progressive nature of polyneuropathy of hereditary transthyretin-mediated amyloidosis, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease."
"Today’s 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.”