# Eplontersen clinical trial and regulatory status

> Live situation record from CLSTR: https://clstr.news/situations/eplontersen-clinical-trial-and-regulatory-status
> Updated: 2026-09-21T13:10:00.000Z. Sources: 35. Developments: 2.

Clinical developments regarding the drug eplontersen have shown divergent results depending on the specific condition being treated. In the phase 3 CARDIO-TTRansform trial, eplontersen failed to significantly reduce cardiovascular mortality or recurrent cardiovascular events in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). While the drug successfully suppressed circulating serum transthyretin levels, it did not meet its primary composite endpoint compared to a placebo. Conversely, the Italian Medicines Agency (AIFA) has approved reimbursement for eplontersen to treat adult patients with stage 1 or 2 hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Based on the phase III Neuro-TTRansform study, the treatment demonstrated a significant reduction in serum transthyretin concentrations and halted the progression of functional impairment related to neuropathy. Specifically, the study indicated an 82% reduction in serum Ttr concentrations at 66 weeks and a significant improvement in patient quality of life. Approved for monthly subcutaneous self-administration via an auto-injector, the drug allows patients to manage treatment at home, potentially reducing the burden on specialized medical centers. During the Salute Direzione Nord event in Milan, Italian health officials reinforced the strategic importance of such treatments. Francesco Saverio Mennini of the Ministry of Health emphasized that investing in prevention, treatment, and early diagnosis—particularly for rare diseases—yields significant returns for both citizen health and the national GDP. In Italy, where the disease affects approximately 600 people annually, officials have noted that such investments may benefit the national economy by reducing productivity losses, estimated at approximately 10,000 euros per year for working patients. Officials also highlighted the need for an integrated approach involving hospitals, local territories, and digitalization to improve access and manage waiting lists. In Lombardy, officials are discussing the use of PNRR resources to transform infrastructure into integrated service models.

## Claims

- Filippo Rumi stated that timely diagnosis, multidisciplinarity, and an appropriate therapeutic pathway are essential for efficient technology access. (corroborated by 3 sources)
- The Italian Medicines Agency (Aifa) has approved the reimbursement of eplontersen. (corroborated by 3 sources)
- Eplontersen is approved for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or 2 polyneuropathy (AttRv-Pn). (corroborated by 3 sources)
- Working patients face an estimated annual productivity loss of approximately 10,000 euros due to the condition. (corroborated by 3 sources)
- Patients travel an average of 53 kilometers to reach specialized treatment centers. (corroborated by 3 sources)

## Timeline

### 2026-09-21: Italy healthcare: Officials advocate for investment and infrastructure expansion

Italian health officials emphasize the economic benefits of medical investment, while Lombardy expands its healthcare infrastructure with a 722.6 million euro hospital hub in Sesto San Giovanni.

33 sources. https://clstr.news/cluster/aifa-approves-eplontersen-for-hereditary-transthyretin-amyloidosis

### 2026-08-28: Eplontersen fails to improve cardiovascular outcomes in ATTR-CM trial

The phase 3 CARDIO-TTRansform trial found that eplontersen failed to significantly reduce cardiovascular mortality or recurrent events in patients with ATTR-CM when added to standard therapy.

2 sources. https://clstr.news/cluster/eplontersen-fails-to-improve-cardiovascular-outcomes-in-attr-cm-trial

---
Cite as: Eplontersen clinical trial and regulatory status. CLSTR, https://clstr.news/situations/eplontersen-clinical-trial-and-regulatory-status