Lonafarnib, also known as Zokinvy, is a drug that is used to minimize the risk of mortality from Hutchinson-Gilford progeria syndrome and to treat specific processing-deficient progeroid laminopathies in persons aged one year and above.
Nausea, vomiting, headaches, diarrhea, infection, decreased appetite, and exhaustion were the most prevalent adverse effects.
In November 2020, Lonafarnib received FDA approval for medicinal use in the United States. It is considered a first-in-class drug by the US Food and Drug Administration (FDA).
Medical applications
In persons one year of age and older, lonafarnib is suggested to minimize the risk of mortality due to Hutchinson-Gilford progeria syndrome and to treat certain additional processing-deficient progeroid laminopathies.
Contraindications
Co-administration of lonafarnib with strong or moderate CYP3A inhibitors and inducers, as well as midazolam and some cholesterol-lowering drugs, is not recommended.
History
Lonafarnib is a farnesyltransferase inhibitor that prevents the accumulation of faulty progerin or progerin-like protein in the body. In 62 individuals from two single-arm studies (Trial 1/NCT00425607 and Trial 2/NCT00916747), the efficacy of lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome was proven when compared to matched, untreated patients from a separate natural history investigation. Patients with Hutchinson-Gilford progeria syndrome who were treated with lonafarnib lived an average of three months longer than untreated patients during the first three years of treatment, and an average of 2.5 years longer throughout the maximal follow-up period of 11 years.
The approval of lonafarnib for the treatment of extremely uncommon processing-deficient progeroid laminopathies was based on parallels in the underlying genetic mechanism of illness and other available evidence. Participants came from 34 different nations, including the United States.
The application for priority review, orphan drug, and breakthrough treatment designations for lonafarnib was approved by the US Food and Drug Administration (FDA). A rare pediatric condition priority review voucher was also given to the manufacturer. Eiger BioPharmaceuticals, Inc. received FDA clearance for Zokinvy.
Research
Lonafarnib is a farnesyltransferase inhibitor (FTI) that has been tested in a human clinical study as a therapy for progeria, a rare genetic condition in which symptoms simulating aging appear at a young age.
Lonafarnib is a tricyclic halogenated carboxamide with anticancer activity that is synthesized. As a result, it's largely utilized to treat cancer. Research has revealed that progeria patients who take the treatment had a lower risk of stroke and transient ischemic attack, as well as a lower prevalence and frequency of headaches. In 2012, a phase II clinical research concluded that a medicine cocktail containing lonafarnib and two additional pharmaceuticals met clinical effectiveness objectives that enhanced the height and reduced the stiffness of progeria patients' bones.
Price
Lonafarnib costs 1556 USD.