New treatment for fragile X syndrome seized FDA orphan drug certification April 04, 2018 Source: Sina Pharmaceutical Recently, clinical drug development company Tetra Discovery Partners announced that the company's new treatment BPN14770 for the treatment of fragile X syndrome has been certified by the US FDA orphan drug. This will further accelerate the development of the drug. BPN14770 is a selective small molecule inhibitor drug that targets phosphodiesterase 4D (PDE4D). The company's previous joint research with the nonprofit organization FRAXA Research Foundation showed that this small molecule drug can significantly improve neuronal function and multiple behavioral indicators in mice in a fragile X syndrome mouse model. Mark E. Gurney, chairman and CEO of the company, said that BPN14770 has the potential to improve a series of cognitive and memory impairments induced by fragile X syndrome and may be applied to autism-related treatments. The company is planning to launch a clinical phase II study of BPN14770 for the treatment of fragile X syndrome in the second quarter of 2018. Previously, BPN14770 had completed three human Phase 1 clinical trials with a total of 147 participants. The results of the study showed that the drug had good safety, high oral bioavailability in elderly subjects and initially determined clinical benefit. Fragile X syndrome is a genetic disorder caused by X-linked fragile X mental retardation-1 (FMR1) gene silencing, and patients exhibit a range of behavioral and other symptoms, including seizures, sleep disorders, anxiety, irritability, ADHD, autism, mild to severe cognitive impairment and mental retardation. Although Fragile X syndrome occurs in both sexes, this condition is more common in men and is usually more severe. The incidence of the disease in men is 1 in 4000, and the incidence in women is 1 in 8000. There is currently no clinically approved treatment for or cure for Fragile X syndrome. Related drugs can only be used to treat symptoms associated with Fragile X syndrome, including seizures or other neuropsychiatric symptoms. Rare diseases generally refer to the type of disease in which the patient has less than 200,000 people. Due to the small number of patients, few pharmaceutical companies have the incentive to develop appropriate drug therapies. Therefore, the development of drug therapy for rare diseases has always been a difficult point in drug development incentive policies in countries around the world. In view of this, the US Food and Drug Administration has awarded orphan drug certification to encourage the development of orphan drugs. The certification allows drug developers to obtain a seven-year US marketing exclusive right and enjoy tax credits for clinical research expenses and apply for annual grant funding, in addition to the FDA's clinical trial design assistance involved in the development of orphan drugs. And reduce the application fee during the drug application process. (Sina Pharmaceutical Compilation / Sheep Pharmacist) Article Reference Source: FDA Grants Orphan Drug Designation for Tetra Discovery Partners' BPN14770 for the Treatment of Fragile X Syndrome Office Safe Box,Commercial Safe Box,Heavy Biometric Office Safe Box,Large Office Safe Box Hebei Yingbo Safe Boxes Co.,Ltd , https://www.ybsafebox.com