国内戈谢病患者迎来国产疗法。 5月16日，北海康成制药有限公司（以下简称"北海康成"）宣布，其全资子公司——北海康成（上海） 生物科技有限公司为持有人的注射用维拉苷酶β（商品名：戈芮宁）获国家药品监督管理局批准上市。 戈芮宁是国内首个本土自主研发适用于12岁及以上青少年和成人Ⅰ型和Ⅲ型戈谢病患者的长期酶替代治 疗，可完全替代同类进口产品。 北海康成创始人、董事长兼首席执行官薛群接受《每日经济新闻》记者采访时表示，相较高昂的进口药 物，戈芮宁可大幅降低患者用药成本。 值得注意的是，据统计，国内戈谢病患者仅约3000人。如何平衡社会意义与企业盈利，是罕见病研发企 业面对的一大难题。为解决这一问题，戈芮宁的CRDMO（合同研究、开发和生产组织）服务公司药明 生物从源头控制罕见病药物的开发成本和生产成本。 药明生物首席执行官陈智胜表示，"在中国开发罕见病药物，一定要通过技术手段，更好地控制成本。 让更多的罕见病药物在比进口产品定价低不少的情况下，仍然具有一定的盈利空间"。 又一罕见病国产疗法获批上市有望大幅降低患者年治疗费用 据悉，戈谢病是最常见的溶酶体贮积症之一，为位于1q22号染色体长臂上的葡萄糖脑苷脂酶基因突变 ...

Summary of PTC Therapeutics Conference Call Company Overview - **Company**: PTC Therapeutics (PTCT) - **Industry**: Biotechnology, focusing on rare diseases and gene therapies Key Points and Arguments Executive Order and Market Position - The recent executive order has unclear ramifications for companies, but PTC Therapeutics expects minimal impact due to its revenue being more international than domestic [4][5] - The company is preparing for the launch of its PKU product, emphasizing a global pricing corridor to maintain product value [5][6] FDA Interactions - PTC has three applications pending with the FDA, with no significant impact from recent agency changes noted [10][12] - The PKU New Drug Application (NDA) is progressing well, with confidence in approval by the PDUFA date [11][12] Translarna Sales in Europe - Despite a negative opinion from the CHMP, PTC is pursuing a country-by-country strategy to commercialize Translarna, with about 50% of countries showing interest [16][17] - The company anticipates achieving 25-30% of European revenue for the year from Translarna [18][20] PKU Launch Strategy - PTC is ready for a global launch of its PKU product, with an early access program initiated in Germany [33][34] - The company has built a robust infrastructure to support the launch, including a patient services team to assist with prior authorizations and patient engagement [38][39] Competitive Landscape for PKU - There are approximately 17,000 individuals with PKU in the US, with a significant unmet need as 85-90% are not on current therapies [44][45] - PTC's PKU therapy is positioned as a better option compared to existing treatments, with a focus on diet liberalization and improved tolerability [47][50] Pricing Strategy - PTC plans to price its PKU therapy at a premium to Palynziq, justified by strong efficacy data and a favorable safety profile [54][55] - The company believes it could achieve a billion-dollar opportunity in the US alone with low double-digit market penetration [57][58] PTC 518 and Huntington's Disease - PTC 518 is positioned as a potential first-ever disease-modifying therapy for Huntington's disease, with promising early data [63][64] - The company is focused on understanding the optimal patient population for this therapy, emphasizing the importance of early-stage patients [66][68] Competitive Landscape for Huntington's Disease - PTC believes that its oral therapy will be more attractive than gene therapies due to its safety, tolerability, and ability to be administered early in the disease process [78][79] - The partnership with Novartis is expected to enhance commercialization efforts for PTC 518 [76][79] Additional Important Content - The company is optimistic about the global opportunity for its therapies, with plans for launches in Japan and other markets [59][61] - PTC is committed to addressing unmet needs in rare diseases, which is a key focus for its product development strategy [32][33]

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CANbridge Pharmaceuticals (01228) H2 2022 Earnings Call March 31, 2023 12:00 PM ET Speaker0 Good morning, everyone. Welcome to Cambridge Pharmaceuticals twenty twenty two twelve Month Annual Results Conference Call. Today, it's a great honor to have Doctor. James Shre, Founder, Chairman and CEO of Cambridge Pharmaceuticals Doctor. Gerald Cox, Chief Medical Officer and Chief Development Strategist Glenn Hassan, Chief Financial Officer and Sophie Hsieh, Director of Investor Relations. Management team will giv ...