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    Home > Active Ingredient News > Drugs Articles > The world's first type 1A treatment drug for Charcot muscular atrophy was approved in domestic clinical trials

    The world's first type 1A treatment drug for Charcot muscular atrophy was approved in domestic clinical trials

    • Last Update: 2018-06-15
    • Source: Internet
    • Author: User
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    Source: Sina pharmaceutical June 14, 2018, Tishi pharmaceutical According to the announcement of the Group Co., Ltd., its holding subsidiary, Tianshili international gene network Pharmaceutical Innovation Center Co., Ltd (hereinafter referred to as "Tianshili gene network company", with a shareholding ratio of 65%), has received the approval of the State Food and Drug Administration (the State Food and Drug Administration) to issue the approval document for drug clinical trials on pxt3003, which will be followed in the future It is required to start the preparation of phase III clinical trials of international multicenter to support Chinese registration The main contents of the clinical trial approval documents are as follows: drug name: pxt3003 approval number: 2018l02544, 2018l02545 dosage form: oral solution specification: 100ml / bottle, according to the concentration of the active ingredient, there are two specifications to apply for: imported drug registration applicant: pharnext main sending unit: tisley international gene network Drug Innovation Center Co., Ltd Acceptance No.: jxhl1800019, Jxhl1800020 registration classification: chemical 2.3 approval conclusion: approve this product for international multi center clinical research According to the announcement, pxt3003 is the world's first drug for the treatment of Charcot Marie Tooth disease type 1A (CMT1A) It is an innovative compound drug that can reduce the expression of PMP22, selected by pharnext company of France from more than 2000 drug molecules that have been on the market by using the biological network pharmacological analysis of CMT1A It is composed of three chemical drugs that have been on the market for many years and fixed proportion at very low dose Tianshili gene network Co., Ltd owns the R & D, production and commercialization rights and interests of pxt3003 project in Greater China (mainland China, Hong Kong, Taiwan and Macao), and exclusive licensing rights and interests of all patents applied for and authorized by pxt3003 in Greater China Pxt3003 project has carried out clinical trials all over the world, exempted phase I clinical studies in Europe, and completed phase II clinical studies of 80 patients The results of phase II clinical effectiveness and safety suggest that pxt3003 has a clear clinical value The international multi center phase III clinical research and development in Europe and the United States is progressing smoothly The intermediate analysis of blind variation analysis and invalid analysis completed in November 2017 has achieved positive results On December 18, 2017, Tianshili gene network company applied to the State Food and Drug Administration for pxt3003 "international multi center phase III clinical trial research to support Chinese registration", which was incorporated into the "priority review procedure" on March 28, 2018, and approved to carry out the international multi center phase III clinical trial research to support Chinese registration on May 28, 2018 Pxt3003 is the world's first drug for the treatment of Charcot muscular atrophy type 1A (CMT1A) CMT1A is a rare peripheral nerve monogenic genetic disease, the patient is suffering and there is no effective treatment and drug Pxt3003 has independent intellectual property rights and has been granted orphan drug status by FDA and EMA (European drug regulatory system) There is obvious genetic heterogeneity in Charcot Marie Tooth disease The incidence rate in Europe and America is 1:3300~1:2500, and CMT1A incidence rate is 57%~37% of CMT There is a lack of epidemiological data in China Up to now, Tianshili gene network company has invested 23.9697 million yuan in pxt3003 project.
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