FDA Approves the First Treatment Intended for Rare Ridging Disease in Children
United States Food and Drug Administration (FDA) approves a medication in the form of a capsule that reduces the risk of deaths caused by Hutchinson-Gilford Progeria syndrome, a rare genetic condition that causes the child's body to age rapidly.
According to Medical Xpress, kids with progeria usually die in their early teens, typically caused by heart disease. FDA added that children with this syndrome and progeroid laminopathies experience accelerated cardiovascular disease from the buildup of defective progerin or progerin-like protein in cells. In the release by US FDA, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine Director in the FDA's Center for Drug Evaluation and Research, Hylton V. Joffe, MD, M.M.Sc. said that Hutchinson-Gilford progeria syndrome and progeroid laminopathies causes premature death and have an incapacitating effect on people's lives. He added in the interview that Zokinvy is the first FDA approved medication for the devastating disease.
The Zokinvy, a treatment taken by mouth, shows promising results in stretching progeria patients' life span.
Medical Xpress furthers that research on the treatment is funded by Progeria Research Foundation located in Peabody, Massachusetts, in collaboration with the Eiger BioPharmacueticals. The Foundation's medical doctor Dr. Leslie Gordon said in Medical Xpress's release that they would find more and better treatments for the disease. In the data by the foundation, as of September 1, 2020, 179 known children are stricken by progeria in 53 countries.
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What is Zokinvy, and what is the method of the study?
According to the US FDA, Zokinvy is a farnesyltransferase inhibitor, taken by mouth, which helps prevent the build-up of faulty progerin or progerin-like protein. They added that the efficacy of zokinvy as a treatment for progeria was shown in 62 patients from two single-arm trials compared to matched, untreated patients from different natural history studies. In their release, FDA notes that the life span of Hutchinson-Gilford progeria syndrome patients treated with Zokinvy heightened by an average of three months in the first three years of administering the drug and an average of 2 and a half years through the maximum follow up of eleven years. This is compared to the patients who did not undergo the treatment.
FDA also highlights in their release that the most common side effects experienced by progeria patients while taking Zokinvy are infection, decreased appetite, fatigue, nausea, vomiting, and diarrhea. They also emphasized that the said drug should not be taken with strong or moderate CYP3A inhibitors and induces some cholesterol-lowering medication, and midazolam, which is commonly used in children before administering anesthetics to cause drowsiness, forget the procedure or surgery, and decrease anxiety. In their release, the FDA furthers that some patients medicated with Zokinvy developed laboratory test abnormalities such as changes in blood potassium and sodium levels, lowered white blood cell counts, and increased liver blood test results.
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FDA's Priority Review Designation
According to the FDA's release, Zokinvy received Orphan Drug designation, which gives incentives to assist and encourage the development of drugs for rare diseases and Breakthrough Therapy Designation. They also added in the release that the manufacturer of Zokinvy was acknowledged for a rare pediatrics disease priority review voucher, which is intended for reassuring the development of new drugs or medication to prevent and treat rare diseases in children.
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Nov 22, 2020 12:00 PM EST
