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
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.
PMID
38548524
DOI
10.1016/j.jfma.2024.03.008
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