The pioneering Frenchi transferase inhibitor zokinvy will be approved for the market

Eiger is a late-stage biopharmaceutical company dedicated to developing and commercializing a range of targeted, pioneering therapies for the treatment of severe and ultra-rare diseases. Recently, the company announced that the U.S. Food and Drug Administration (FDA) has accepted a new drug application (NDA) from Lonafarnib. The NDA seeks to accelerate approval of Zokinvy for the treatment of premature aging (Progeria, also known as Hudkinson-Guildford early aging syndrome, HGPS) and premature aging-like nuclear fibrinosis (Progeroid Laminopathies). The FDA has granted Zokinvy NDA priority review and designated the target date for the Prescription Drug User Charge Act (PDUFA) as November 20, 2020. The FDA has informed Eiger that it does not plan to convene an advisory committee meeting to discuss the NDA.If approved, Zokinvy would be the world's first drug to treat premature aging. Zokinvy is also undergoing an accelerated assessment by the European Medicines Agency (EMA). Previously, Zokinvy was awarded orphan drug eligibility (ODD), breakthrough drug eligibility (BTD) and rare pediatric disease (RPDD) by the FDA and the European Union EMA for the treatment of premature aging and early aging-like nuclear fibrinogen disease.Premature aging is an extremely rare and deadly disease that can lead to premature aging in children. If untreated, premature aging children will die of heart disease, with an average age of 14.5 years. At present, there is no approved treatment for premature aging and premature aging-like nuclear fiber protein disease.Zokinvy's active drug ingredient is lonafarnib, a pioneering oral niki 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 early aging, lonafarnib single-drug therapy reduced the risk of death by 88 percent, with the most common adverse reaction reported to be gastrointestinal symptoms. Many patients with premature aging have been receiving continuous treatment at lorafarnib 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 premature aging and premature aging-like nuclear fiber protein disease have access to treatment.David Cory, President and CEO of Eiger, said, "The FDA's acceptance of our first NDA is an important milestone for Eiger and an important step toward the treatment of children and young people with advanced early aging and progressive early aging-like nuclear fiber protein disease. We would like to thank the Early Aging Research Foundation (PRF) for its commitment, persistence and dedication. Most importantly, we thank all children and their families who have made this possible by participating in the Lorafarnib clinical trial. We are preparing for Zokinvy's commercial launch in the United States and Europe. Leslie Gordon, M.D., medical director of PRF, said: "This milestone is the culmination of 12 years of clinical trials that have treated children with premature aging and premature aging-like nuclear fiber protein disease in more than 30 countries and six continents around the world. We are fortunate to be a partner of Eiger in the preparation and submission of NDA and in providing ongoing Zokinvy supply for children and young people with early aging. We thank all the children and their families who suffer from premature aging. Their courage inspires us every day. Early(HGPS) is a rare and deadly genetic disorder that accelerates aging in children. The disease is caused by a point mutation in the LMNA gene that encodes the nuclear fiber protein A (lamin A), which produces an abnormal, toxic premature aging protein (progerin). The nuclear fiber layer protein A is part of the cellular structure stent and plays an important role in the structure and function of the nucleoblast. Children with early aging die from the same disease that affects millions of normal aging adults, atherosclerosis, but the average age of death is 14.5 years. Symptoms of premature aging include severe stunting, sclerosis-like skin, systemic fat malnutrition, hair loss, joint contractions, bone dysplation, accelerated atherosclerosis throughout the body, decreased cardiovascular function, and stroke. Globally, it is estimated that there are about 400 cases of premature aging in children.Premature aging-like nuclear fiber protein disease (progeroid laminopathies) is the inheritance of accelerated aging caused by a series of mutations in the nucleosynin A (lamin A) and/or Zmpste24 genes that produce fanylated proteins that differ from premature aging proteins. Although no premature aging proteins are produced, these genetic mutations can lead to overlapping but differentiated disease manifestations with premature aging proteins. In general, globally, the incidence of premature aging-like nuclear fiber protein disease may be higher than that of premature aging.Lonafarnib is a distinctive, late-development oral activity inhibitor targeted by a frenchiki transferase, which is involved in the modification of proteins through a process called isoprene. Premature aging protein is an abnormal protein of frenchylization that is thought not to be cut, resulting in tight binding to the nuclear membrane, leading to changes in the shape of the nucleofilm and subsequent cell damage.Lonafarnib blocks the early-oncourse protein, and more than 90 children with early-aging were treated in Phases I/II and II studies funded by the Research.org Foundation for Early Aging at Boston Children's Hospital. The results of the two studies showed that after 2.2 years of follow-up, the mortality rate of children with lonafarnib single-drug treatment was lower (3.7% vs. 33.3%; HR was 0.12) and the risk of death was 88% lower than that of untreated children with early aging.In addition to treating premature aging and premature aging-like nuclear fiber protein disease, Eiger is also developing lolafarnib to treat hepatitis D virus (HDV) infections. At the end of December 2018, the FDA and EMA granted lomafarnib breakthrough drug eligibility (BTD) and priority drug eligibility (PRIME) for the treatment of hepatitis D virus infection (HDV), respectively. Lonafarnib inhibits isoprene steps for HDV replication in liver cells, blocking the virus life cycle during the virus assembly phase.Data from multiple Phase II clinical studies confirm the efficacy and safety of the lonafarnib-based program for treating HDV infections, and the data show that among HDV-infected people who have not previously been treated (primary treatment), the lonafarnib-based program achieves a combination of reduced HDV RNA levels ≥2log10 and normalization of alanine amino transferase (ALT), reflecting improvements in liver condition and viral response. Currently, the drug is in the midst of a critical HPV Phase III clinical study.Hepatitis D is caused by HDV infection and is the most serious type of human viral hepatitis. Hepatitis B occurs only as a co-infection in individuals with the hepatitis B virus (HBV), causing liver disease that is more severe than hepatitis B and accelerating liver fibrosis, liver cancer and liver failure. Hepatitis D is a disease that seriously affects human health worldwide and can affect about 15 to 20 million people worldwide. Hepatitis D is endemic in different parts of the world. Globally, about 4.3%-5.7% of people with chronic hepatitis B virus are reported to have HDV infection. In some regions, including Outer Mongolia, China, Russia, Central Asia, Pakistan, Turkey, Africa, the Middle East and South America, the prevalence of HDV among people infected with chronic HBV is even higher, with HDV prevalence reported to be as high as 60% (Bio Valley Bioon.com) in Mongolia andPakistan