The world's first treatment for premature aging has been approved by the FDA
-
Last Update: 2021-03-05
-
Source: Internet
-
Author: User
Search more information of high quality chemicals, good prices and reliable suppliers, visit
www.echemi.com
recently, Eiger announced that its ZokinvyTM (lonafarnib) has been approved by the U.S. FDA to treat patients 12 months and older with early aging syndrome (HGPS or premature aging) and premature aging-like nuclear fiber protein disease (PL). This is the first FDA-approved drug to treat premature aging.Early aging, also known as Hutchinson-Gilford Progeria syndrome, or early childhood aging, is a genetic disorder. Sick children's bodies age 5 to 10 times faster than normal, patients look like the elderly, organs rapidly decline, resulting in a decline in physiological function. Sick children generally live to 7 to 20 years of age, most of them will die from aging diseases, such as cardiovascular disease, and there is no effective treatment, can only rely on drugs for treatment.Lonafarnib is a pioneering oral frenchiki transferase inhibitor (FTI) that blocks the fatnikidification of premature aging proteins and has shown statistically significant survival benefits in children and young people with premature aging. An observational queue study assessed mortality rates in children with early aging who received lorafarnib monodrive therapy (n s 63) and in children with premature aging (n s 63) who were not treated with lorafarnib. These children were matched by sex, age and area of residence to those who were not treated. The dose treated with lorafarnib monotherapy is 150 mg / m2 twice daily.In this analysis, 4 deaths (6.3%) were reported in 63 patients treated with Lonafarnib, while 17 deaths (27%) were in the untreated pairing group. Compared to the untreated control group, children treated with Lorafarnib had a 77 per cent reduction in mortality during the follow-up period of an average of 2.2 years (risk ratio 0.23; P s 0.04). The results were published in the 2018 issue of the Journal of the American Medical Association (JAMA).In five Phase 1/2 and Phase 2 studies, more than 90 children with early aging took Lorafarnib. The drug is well-resistant and the most common adverse reactions occur in the gastrointestinal tract, including vomiting, diarrhea and nausea, but most are mild or moderate (level 1 or 2) and side effects can be well controlled through preventive treatment of laxatives and anti-spitting drugs. Many premature aging patients have been receiving Zokinvy treatment continuously for more than 10 years.Lonafarnib is understood to have been granted the "orphan drug" status for premature aging by the FDA and the European Medicines Agency (EMA), as well as fast-track and breakthrough drug eligibility from the FDA, as well as the EMA's Priority Drug Qualification (PRIME).Eiger is a late-stage biopharmaceutical company dedicated to developing and commercializing best-in-class treatments for serious and ultra-rare diseases. In addition to being used to treat premature aging and premature aging-like nuclear fiber protein disease, lonafarnib's main clinical program targets the most severe form of human viral hepatitis, hepatitis buttitis (HDV) infection.HDV occurs as a co-infection only in individuals infected with the hepatitis B virus (HBV). HDV causes more serious liver disease than individual HBVs and is associated with accelerated liver fibrosis, liver cancer and liver failure. Approved HBV nucleotide therapy can only inhibit HBV DNA, does not affect HBsAg (hepatitis B surface antigen) and does not affect HDV.Lonafarnib is the first and only HDV oral drug under development. HDV uses the host cell process inside the liver cell to complete key steps in its life cycle, while Lonefarnib inhibits the isopreneization step that HDV replicates inside the liver cell and blocks the life cycle of the virus during assembly. It is reported that the drug is currently in a key HDV Phase III clinical study. (Biological Exploration):
This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only.
This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of
the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed
description of the concern or complaint, to
service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content
will be removed immediately.