The US Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) for eplontersen, a novel medicine for the treatment of patients with polyneuropathy linked to hereditary transthyretin amyloidosis (hATTR), Ionis Pharmaceuticals announced.

The application has been granted a Prescription Drug User Fee Act action date of December 22, 2023. The FDA did not make any additional data requests, stated there were no review issues, and said it has no plans to hold an advisory committee meeting to discuss the application, according to a press release from Ionis.

The company previously conducted the phase 3 NEURO-TTRansform study which analyzed the effectiveness of the medication in hATTR patients with polyneuropathy and compared the results with a group receiving a placebo.

“Significant reductions in transthyretin (TTR) protein levels were observed during the NEURO-TTRansform 35-week interim analysis,” said Eugene Schneider, MD, executive vice president and chief clinical development officer at Ionis. “The interim analysis demonstrated eplontersen has the potential to make a positive impact on disease progression and improve quality of life in a substantial number of patients,”

According to the study results, treatment with eplontersen resulted in a significant mean reduction of serum TTR concentration compared with baseline levels. The results of the modified Neuropathy Impairment Score +7 also showed a beneficial effect on neuropathic disease progression.

Read more about hATTR complications

Moreover, the researchers revealed hATTR patient-reported quality of life improvements in comparison with baseline levels, obtained by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy. The safety and tolerability profile of eplontersen was evaluated as satisfactory.

The medication previously received the FDA’s Orphan Drug Designation for hATTR and is being evaluated in the global phase 3 CARDIO-TTRansform study for hATTR-related cardiomyopathy.

Eplontersen is an experimental therapy developed by Ionis and AstraZeneca for the treatment of both hereditary and nonhereditary forms of hATTR. By reducing the production of TTR, the medication helps with debilitating nerve damage and progressive loss of motor function caused by TTR accumulation in bodily organs.


Ionis announces FDA acceptance of new drug application for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). News release. Ionis Pharmaceuticals; March 07, 2023.