Summary

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• Lonafarnib (Zokinvy) is indicated for 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, as well as for treatment of processing-deficient progeroid laminopathies with heterozygous LMNA mutation or homozygous/compound heterozygous ZMPSTE24 mutations.
• The information was derived from Randomized Controlled Trials.
• In terms of safety, no participants withdrew due to side effects during the trial, which indicates that lonafarnib has a favorable safety profile when used alone or in combination with pravastatin and zoledronic acid.
• For effectiveness, primary outcomes showed improvement in weight gain rate or carotid artery echodensity in 71% of participants; secondary improvements were observed mainly in bone mineral density but not significantly in cardiovascular benefits beyond what is achieved by lonafarnib monotherapy.
• Compared to other treatments, specifically pravastatin and zoledronic acid, when used together with lonafarnib, the only additional benefit was seen in bone mineral density, while improvements in cardiovascular benefits did not exceed those made by the lone use of lonafarnib.
• The study focused exclusively on patients suffering from Hutchinson-Gilford Progeria Syndrome without detailing any subgroup considerations beyond age criteria and body surface area required for treatment eligibility.