Summary

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• Zokinvy (lonafarnib) is indicated in patients 12 months of age and older with a body surface area of 0.39 m² and above to reduce the risk of mortality in Hutchinson-Gilford progeria syndrome. It is also indicated in patients 12 months of age and older with a body surface area of 0.39 m² and above for the treatment of processing-deficient progeroid laminopathies with heterozygous LMNA mutation with progerin-like protein accumulation, as well as for the treatment of processing-deficient progeroid laminopathies with homozygous or compound heterozygous ZMPSTE24 mutations.
• This summary is based on the review of one randomized controlled trial(s). ^[1]
• Primary Outcome: In participants with Hutchinson-Gilford progeria syndrome, 71.0% met the predefined primary outcome of improved weight gain or carotid artery echodensity (P<0.0001).
• Secondary Outcomes: Significant improvements were observed in areal bone mineral density (P=0.001) and volumetric bone mineral density (P<0.001-0.006), with a 1.5- to 1.8-fold increase in radial bone structure (P<0.001).
• Comparison to Monotherapy: The combination therapy provided additional benefits in bone mineral density but did not show significant improvements in cardiovascular outcomes, such as carotid artery wall echodensity and carotid-femoral pulse wave velocity, compared to lonafarnib monotherapy.
• No participants withdrew from the trial due to side effects. However, the percentage of participants with carotid artery plaques increased from 5% to 50% (P=0.001), femoral artery plaques increased from 0% to 12% (P=0.13), and extraskeletal calcifications increased from 34.4% to 65.6% (P=0.006).
• There is no population types or subgroups information available in the reviewed studies.