Drug updated on 12/11/2024
| Dosage Form | Capsule (oral; 50 mg, 75 mg) |
| Drug Class | Farnesyltransferase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated in patients 12 months of age and older with a body surface area of 0.39 m and above to reduce risk of mortality in Hutchinson-Gilford progeria syndrome
- Indicated in patients 12 months of age and older with a body surface area of 0.39 m and above for treatment of processing-deficient progeroid laminopathies with heterozygous LMNA mutation with progerin-like protein accumulation
- Indicated in patients 12 months of age and older with a body surface area of 0.39 m and above for treatment of processing-deficient progeroid laminopathies with homozygous or compound heterozygous ZMPSTE24 mutations.
Latest News
Summary
- This summary is based on the review of one randomized controlled trial. [1]
- Participants achieved a 71.0% rate success in the primary outcome of weight gain or carotid artery echodensity (P<0.0001).
- Areal and volumetric bone mineral density increased significantly (P=0.001; P<0.001–0.006), with 1.5- to 1.8-fold increases in radial bone structure (P<0.001), exceeding the benefits observed with lonafarnib monotherapy alone.
- No significant changes were observed in carotid artery wall echodensity or pulse wave velocity. Carotid artery plaques increased from 5% to 50% (P=0.001), femoral artery plaques from 0% to 12% (P=0.13), and extraskeletal calcifications from 34.4% to 65.6% (P=0.006), with no additional cardiovascular benefits from the combination therapy over lonafarnib monotherapy.
- No participants withdrew from the trial due to side effects.
- Specific adverse effects or safety concerns were not listed in the study.
- There is no population types or subgroups information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Zokinvy (lonafarnib) Prescribing Information. | 2024 | Eiger BioPharmaceuticals, Inc., Palo Alto, CA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Clinical Trial of the Protein Farnesylation Inhibitors Lonafarnib, Pravastatin, and Zoledronic Acid in Children With Hutchinson-Gilford Progeria Syndrome. | 60Subjects F: 58% M: 42% | 2016 | Circulation |
Sex Distribution:
F:58%
M:42%
60Subjects
Year:
2016
Source:Circulation