The world's first early-aging drug! Zokinvy, the pioneering Faniki transfer enzyme inhibitor, will be approved for the market in November, reducing the risk of death by 88 percent!

Early Aging Boys - Berns, who died at age 17
May 20, 2020 /
BioValley
BIOON/--Eiger is a late-stage biopharmaceutical company dedicated to the development and commercialization of a range of targeted, pioneering therapies for the treatment of severe and ultra-rare diseasesThe company recently announced that the U.SFood and Drug Administration (FDA) has accepted zokinvy's application for a new drug (NDA)The NDA sought accelerated approval of Zokinvy for the treatment of premature anterior disease (Progeria, also known as Hutchinson-Guildford premature aging syndrome, HGPS) and premature aging-like nucleofibrosis (Progeroid Laminopathies)The FDA has granted ZokinvyNDA priority review and has set a target date for the Prescription Drug User Charge Act (PDUFA) as November 20, 2020FDAhas informed Eiger that it does not plan to convene an advisory committee to discuss the NDAmeetingif approved, Zokinvy would become the world's first drug to treat premature agingZokinvy is also undergoing an accelerated assessment by the European Medicines Agency (EMA)Previously, Zokinvy has been awarded the U.SFDA and the European Union EMA for orphan drug eligibility (ODD),FDAgranted breakthrough drug eligibility (BTD) and rare pediatric disease eligibility (RPDD) premature aging is an extremely rare and fatal disease that can lead to premature aging in children If untreated, children with premature anetics die from heart disease, with an average age of 14.5 years Currently, there are no approved treatments for premature aging and premature aging-like nuclear fibrosis Zokinvy's active pharmaceutical ingredient is lonafarnib, a pioneering oral fanny transferase inhibitor (FTI) that has been shown to extend survival in children with premature aging and young adults A 2018 study published in the Journal of the American Medical Association (JAMA) found that in patients with premature aging, lonafarnib monotherapy reduced the risk of death by 88 , with the most common adverse reactionreported being gastrointestinal symptoms Many patients with premature aging have been receiving lonafarnib for continuous treatment for more than 10 years Eiger has also established a global management access program, which is expected to cover more than 40 countries, to ensure that all children and young people with early-aging and premature-like rifiplasmia sitoritis have access to treatment "
," said David Cory, president and chief executive officer of Eiger FDA accept our first NDA is an important milestone for Eiger and an important step toward treating children and young people with progressive premature aging and progressive premature aging of nuclear fibrosis We would like to thank the Early Aging Research Foundation (PRF) for its commitment, commitment and dedication Most importantly, we thank all children and their families with premature aging, who have made this possible by participating in the clinical trials of lonafarnib
We are preparing for Zokinvy's commercial launch in the United States and Europe "This milestone is the culmination of 12 years of clinical trials treating children with premature aging and premature nucleofia from more than 30 countries and six continents around the world," said Leslie Gordon, M.D., medical director, PRF We are fortunate to be a partner with Eiger in the ndA preparation and submission, providing continuous Zokinvy supply to children and young people with premature aging We thank all the children and their families who suffer from premature aging Their courage inspires us every day lonafarnib molecular structure (Photo: Wikipedia) premature aging (HGPS) is a rare and fatal childhood accelerated aging genetic disease The disease is caused by a mutation in the LMNA gene that encodes the nucleofibre protein A (laminA), which produces a fanikin abnormal, toxic early aging protein (progerin) Nuclear fibroid protein A is a part of the nucleostructural stent of the cell and plays an important role in the structure and function of the nucleus Children with premature aging die from the same disease that affects millions of normally ageing adults - atherosclerosis, but the average age of death in children is 14.5 years Early aging diseases include severe stunting, sclerecarina-like skin, systemic fat malnutrition, hair loss, joint contraction, bone dysplasia, accelerated atherosclerosis throughout the body, decreased cardiovascular function, and stroke Globally, there are an estimated 400 cases of premature aging in children early-aging-like nucleofibre protein disease (progeroidlaminopathies) is a genetic of accelerated aging caused by a series of mutations in the nuclear fibrous protein A (laminA) and/or Zmpste24 genes, which produce a fanichemical protein that is different from the early-aging protein Although early-onstage proteins are not produced, these gene mutations can lead to disease manifestations that overlap with premature aging proteins Overall, the incidence of early-aging-like nuclear fibrosis may be higher globally than early aging lonafarnib is an oral active inhibitor with distinctive characteristics and in the late stages of development, targeting the Faniki transfer enzyme, which is involved in protein modification through a process called isopreneization Premature aging protein is a faniki-chemicalabnormal protein that is considered to be uncut, leading to a close bond with the nuclear membrane, leading to changes in the shape of the membrane and subsequent cell damage lonafarnib, which blocks the use of premature aging proteins, treated more than 90 children with premature aging in phaseie I/II and II studies funded by the Boston Children's Hospital Foundation for Early Aging Research ( The results of the two studies showed that after 2.2 years of follow-up , the infant mortality rate of premature aging (3.7% vs33.3 per cent; HR s 0.12) and the risk of death decreased by 88 per cent compared to children with untreated premature anjaculator In addition to treating premature aging and premature nucleofia, Eiger is also developing lonafarnib to treat hepatitis D virus (HDV) infection At the end of December 2018, FDA and EMA granted lonafarnib the Breakthrough Drug Qualification (BTD) and Priority Drug Qualification (PRIME) for the treatment of hepatitis C virus infection (HDV), respectively Lonafarnib inhibits the isopreneization of HDV replication in liver cells, blocking the virus life cycle during the virus assembly phase data from a number of Phase II clinical studies confirmed the efficacy and safety of lonafarnib-based treatment of HDV infections, showing that lonafarnib-based solutions reached a joint primary endpoint that reduced HDVRNA levels by 2log10 and normalized asalanic acid (ALT), reflecting improved liver conditions and virological responses At present, the drug is in a key phase of HDVIII clinical research hepatitis D is caused by HDV infection and is the most serious type of viral hepatitis in humans Hepatitis D occurs only as co-infection in individuals carrying the hepatitis B virus (HBV), causing more severe liver disease than hepatitis B, and accelerates liver fibrosis, liver cancer
and liver failure Hepatitis D is a disease that seriously affects human health worldwide and affects about 15 to 20 million people worldwide The prevalence of hepatitis D varies from region to region around the world Globally, about 4.3%-5.7% of people living with chronic hepatitis B virus are reported to have HDV infection In some regions, including Mongolia, China, Russia, Central Asia, Pakistan, Turkey, Africa, the Middle East and South America, the prevalence of HDV in people infected with chronic HBV is even higher, with the prevalence of HDV reported to be as high as 60% in people infected with HBV in Mongolia and Pakistan (BioValley Bioon) .com) original origin: EigerBioPharmaceuticals Announces
FDA AcceptanceofNDAforFiling Priority Review of Zokinvy (lonafarnib) for Treatment of Progeriaand Progeroid Laminopathies