AstraZeneca Submits NDA in China for Once-Monthly Self-Administered ASO Therapy Eplontersen

Today (September 29), the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has just announced,AstraZeneca's marketing application for eplontersen injection, filed under Registration Category 5.1, has been accepted.Public information shows that this is AstraZeneca and Ionis PharmaceuticalsA jointly developed, once-a-month, self-administered subcutaneous injection of antisense oligonucleotide (ASO) therapy, which was approved for marketing by the U.S. FDA in December last year, treatingAdult Hereditary Transthyretin (TTR) Mediated Amyloidosis with Polyneuropathy (ATTRv-PN)。

Screenshot source: CDE official website

The single-stranded DNA molecules of ASO drugs can lead to mRNA degradation or alter mRNA splicing by binding to specific RNA sequences, thereby achieving the effect of targeting the underlying mechanisms of diseases at the mRNA level to treat diseases.

Eplontersen InjectionIs adopting IonisAn ASO drug developed using the company's Ligand-Conjugated Antisense Oligonucleotide (LICA) technology.It is designed for once-a-month administration and can be self-administered subcutaneously by the patient.Administered by the patient using an autoinjector. The productBy inhibiting the production of TTR protein, thereby treating hereditary and non-hereditary amyloidosis polyneuropathy。

According toIonisAccording to the company's public information,The LICA technology platform can improve the characteristics of ASO drugs in various ways by conjugating different small molecules to ASOs., not only allowing them to be administered through various methods, but also enabling delivery to different organs and tissues in the human body.In December 2021, AstraZeneca announced a global development and commercialization agreement with Ionis for eplontersen.Agreement, thereby jointly developing the product.

In December 2023, eplontersen received FDA approvalApproved for Marketing, used to treat adults with hereditary transthyretin (TTR)-mediated amyloidosis polyneuropathy (ATTRv-PN). The efficacy of this product was demonstrated in a randomized, open-label, multicenter Phase 3 clinical trial conducted in adult patients with ATTRv-PN. Analysis showed that at 35 weeks of treatment, compared with the external placebo control,eplontersenTreatment led to statistically significant improvements in patients' modified Neuropathy Impairment Score +7 (mNIS+7) and total score of the quality of life metric (Norfolk QoL-DN).。

Screenshot source:China Drug Clinical Trial Registration and Information Disclosure Platform

In addition to treatmentATTRv-PN, researchers are still developingePlontersen Injection TreatmentTransthyretin Amyloid Cardiomyopathy (ATTR-CM).According to the official website of the Chinese Drug Clinical Trial Registration and Information Disclosure Platform, AstraZeneca has launched aPhase 3 Clinical Study in China, aiming toChinese ATTR-CM SubjectsEvaluate the effect of eplontersen on transthyretin (TTR) reduction and its long-term safety.

This timeEplontersen's submission for marketing approval in China marks a significant advancement in the product's progress within the country.