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Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study

Publication Details

Journal of the Formosan Medical Association

March 2024

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Author(s)

Kon-Ping Lin¹, Chih-Chao Yang², Yi-Chung Lee¹, Ming-Jen Lee², John Vest³, Marianne T Sweetser³, Matthew T White³, Prajakta Badri³, Sung-Tsang Hsieh², Chi-Chao Chao⁴

Affiliations

¹Department of Neurology, Taipei Veterans General Hospital, Taipeo, Taiwan; ²Department of Neurology, National Taiwan University Hospital, Taipei, Taiwan; ³Alnylam Pharmaceuticals, Cambridge, MA, USA; ⁴Department of Neurology, National Taiwan University Hospital, Taipei, Taiwan

Abstract

Background:

To examine the efficacy and safety of patisiran, an RNA interference therapeutic, in patients from Taiwan with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy.

Methods:

The APOLLO phase 3 trial included patients from Taiwan who received patisiran 0.3 mg/kg intravenously or placebo once every 3 weeks (q3w) for 18 months (18 M), followed by patisiran 0.3 mg/kg q3w in an ongoing global open-label extension (OLE) study. The primary endpoint was change from baseline in modified Neuropathy Impairment Score +7 (mNIS+7) at 18 M.

Results:

Eighteen Taiwanese patients were enrolled in APOLLO (patisiran, n = 8; placebo, n = 10; all A97S gene variant) and 14 continued in the global OLE. In this Taiwanese sub-population, beneficial treatment effects at 18 M were observed in mNIS+7 (least squares mean difference in change from baseline [patisiran-placebo], −26.5 points; 95% confidence interval: −45.5, −7.5). Patients who switched from placebo to patisiran demonstrated slowing of polyneuropathy progression at month 12 in the global OLE, while those who received patisiran in APOLLO maintained the beneficial treatment effects. Patisiran had an acceptable safety profile in the Taiwanese sub-population.

Conclusions:

This analysis suggests that patisiran is well tolerated and may provide a substantial clinical benefit for Taiwanese patients with hATTR amyloidosis with polyneuropathy.