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Eplontersen clinical trial and regulatory status

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2026-09-21 19:41 UTC → 2026-09-21 21:05 UTC · added ~~removed~~

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.

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