Ultragenyx submits NEW drug application to UX007 (triheptanoin) to FDA

LC-FAOD is a group of autosomal recessive genetic diseases characterized by metabolic defects in the body's inability to convert long-chain fatty acids into energyFailure to generate energy from fat can lead to severe consumption of glucose in the body, leading to acute metabolic crises, such as common infections or moderate exercise, as energy demand increasesRecently, UltragenyxCompany(http:// announcedthat it had submitted to the u.SFood andDrug(http://Administration (
FDA(http://) aapplication (NDA) for theof the new http:// drug(triheptanoin, trichomoniase triglycerides), or glycerides, or glycec triglycerides 　　About UX007
UX007 is a high-purity, medicine (http:// grade, medium-chain triglycerides that connect three hexacarbon fatty acids to the synthetic (http:// the glycotic acid triglycerides produced on the glycerin skeleton through a multi-step chemical process 　　UX007 is designed to provide patients with medium-length odd-chain fatty acids that can be metabolized in the Key Energy Generation Process Krebs Cycle to increase intermediate substrates, thus directly addressing LC-FAOD defects 　　The submission of UX007 NDA was supported by a comprehensive package, including a Phase II study from 29 patients enrolled in the group, a long-term safety and efficacy extension study of 75 patients in the group (including 20 patients with initial treatment of UX007), a retrospective medical (http:// review of 20 patients who initially received the drug, and a randomized study of 32 patients