Bioprojet Pharma SARL

Phase 2 signal detection study presented at the annual meeting of the Associated Professional Sleep Societies, "SLEEP 2024" [05-June-2024] PLYMOUTH MEETING, Pa., June 5, 2024 /PRNewswire/ -- Harmony Biosciences (Nasdaq: HRMY) presented data from its Phase 2 signal detection study showing that pitolisant reduced excessive daytime sleepiness (EDS) and fatigue in adults with Myotonic dystrophy Type 1 (DM1). "More than 80 percent of DM1 patients experience EDS and fatigue, which patient-reported outcomes research has shown to be nearly as debilitating as the primary symptoms of DM1, namely myotonia and muscle weakness,"1 said Kumar Budur, M.D., M.S., Chief Medical and Scientific Officer of Harmony Biosciences. "The findings from our signal detection study evaluating pitolisant, which is believed to promote wakefulness through histamine, present an exciting opportunity to develop new treatments for EDS and fatigue in DM1, narcolepsy, and the other sleep/wake disorders we are investigating through our lifecycle management programs that will involve Next-Generation formulations of pitolisant." Notably, there was greater mean improvement from baseline to Week 11 in both EDS (as measured by the Daytime Sleepiness Scale) and fatigue (as measured by the Fatigue Severity Scale) compared to placebo. The overall disease burden also signaled greater improvement for pitolisant compared to placebo, with the higher dose pitolisant group showing a stronger efficacy signal. This study was designed for signal detection and was not powered to demonstrate statistical significance. Estimates suggest there are 40,000 people currently diagnosed with DM1 in the U.S. with up to 90% of them reporting EDS and fatigue. "Given the potential opportunity of pitolisant for treating EDS and fatigue in patients with DM1, we plan to progress our DM1 development program through a pivotal Phase 3 study using the Next-Generation 2 (NG2) formulation of pitolisant, which is designed to deliver an optimized pharmacokinetic pro higher dosage strength," Budur added. "The NG2 formulation could be promising given the positive findings we observed across the EDS and fatigue endpoints within the higher dose pitolisant treatment arm. These efforts are integral to our broader life cycle management programs, which, if successful, could benefit over 100,000 patients living with unmet medical needs." Poster 378: Primary Efficacy and Safety Results of a Phase 2, Double-Blind, Placebo-Controlled, Proof of Concept, Signal Detection Study of Pitolisant in Myotonic Dystrophy The Phase 2 signal detection study was a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of pitolisant in the treatment of EDS and other non-muscular symptoms in adults with DM1. Eligible patients were confirmed to have DM1 and enrolled in an 11-week double-blind treatment phase that included a 3-week titration period and an 8-week stable dose period. Participants were randomized to receive higher or lower dose pitolisant, or a matching placebo. The primary efficacy endpoint was the change from baseline to Week 11 in Daytime Sleepiness Scale score. Additional efficacy endpoints included the change from baseline to Week 11 in Epworth Sleepiness Scale score, Fatigue Severity Scale score, Clinical Global Impression of Severity (for EDS) score, and Myotonic Dystrophy Health Index score. Results from the study include: Mean improvement on the Daytime Sleepiness Scale (primary endpoint) was greater for pitolisant. Compared with placebo, a dose-response relationship was observed from baseline to Week 11: Higher Dose Pitolisant (n=8): -2.5 Lower Dose Pitolisant (n=8): -1.0 Placebo (n=9): -0.2 Mean improvement on the other secondary efficacy endpoints (EDS, fatigue, disease burden) was also greater for pitolisant versus placebo, with higher dose pitolisant showing a stronger efficacy signal from baseline to Week 11. Epworth Sleepiness Scale: Higher Dose Pitolisant (n=8): -4.9 Lower Dose Pitolisant (n=9): 1.3 Placebo (n=10): -0.1 Fatigue Severity Scale: Higher Dose Pitolisant (n=8): -0.9 Lower Dose Pitolisant (n=9): -0.4 Placebo (n=10): -0.1 Clinical Global Impression of Severity (for EDS): Higher Dose Pitolisant (n=8): -0.9 Lower Dose Pitolisant (n=9): -0.2 Placebo (n=10): -0.1 Myotonic Dystrophy Health Index Higher Dose Pitolisant (n=8): -9.1 Lower Dose Pitolisant (n=9): -2.9 Placebo (n=10): 0.4 The rate of adverse events was similar for pitolisant and placebo. The safety and tolerability of pitolisant in patients with DM1 were consistent with its known safety profile. Pitolisant is marketed as WAKIX® in the U.S. and is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy. Pitolisant is not approved for use in patients with DM1 and is currently being evaluated as an investigational agent in this patient population. 1 Heatwole C, Bode R, Johnson N, et al. Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1). Neurology. 2012; 79(4):348-57. Erratum in: Neurology. 2012; 79(13):1411. About Myotonic Dystrophy Type 1 Myotonic dystrophy Type 1 (DM1) is the most common form of adult-onset muscular dystrophy. It is a genetic disorder inherited in an autosomal-dominant pattern. Latest estimates suggest a prevalence of about one per 2,100 people with the genetic defect for DM1. This equates to about 150,000 people in the U.S. with the genetic defect for DM1. About WAKIX® (pitolisant) Tablets WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and has been commercially available in the U.S. since Q4 2019. It was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States. Indications and Usage WAKIX is indicated for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. Important Safety Information Contraindications WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment. Warnings and Precautions WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD). Adverse Reactions In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and at least twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at ≥2% and more frequently than in patients treated with placebo included headache, upper respiratory tract infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash. Drug Interactions Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half. Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required (see full prescribing information). H1 receptor antagonists that cross the blood-brain barrier may reduce the effectiveness of WAKIX. Patients should avoid centrally acting H1 receptor antagonists. WAKIX is a borderline/weak inducer of CYP3A4. Therefore, reduced effectiveness of sensitive CYP3A4 substrates may occur when used concomitantly with WAKIX. The effectiveness of hormonal contraceptives may be reduced when used with WAKIX and effectiveness may be reduced for 21 days after discontinuation of therapy. Use in Specific Populations WAKIX may reduce the effectiveness of hormonal contraceptives. Patients using hormonal contraception should be advised to use an alternative non-hormonal contraceptive method during treatment with WAKIX and for at least 21 days after discontinuing treatment. There is a pregnancy exposure registry that monitors pregnancy outcomes in women who are exposed to WAKIX during pregnancy. Patients should be encouraged to enroll in the WAKIX pregnancy registry if they become pregnant. To enroll or obtain information from the registry, patients can call 1-800-833-7460. The safety and effectiveness of WAKIX have not been established in patients less than 18 years of age. WAKIX is extensively metabolized by the liver. WAKIX is contraindicated in patients with severe hepatic impairment. Dosage adjustment is required in patients with moderate hepatic impairment. WAKIX is not recommended in patients with end-stage renal disease. Dosage adjustment of WAKIX is recommended in patients with moderate or severe renal impairment. Dosage reduction is recommended in patients known to be poor CYP2D6 metabolizers; these patients have higher concentrations of WAKIX than normal CYP2D6 metabolizers. Please see the Full Prescribing Information for WAKIX for more information. To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088 or . About Harmony Biosciences At Harmony Biosciences, we specialize in developing and delivering treatments for rare neurological diseases that others often overlook. We believe that where empathy and innovation meet, a better life can begin for people living with neurological diseases. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, PA, our team of experts from a wide variety of disciplines and experiences is driven by our shared conviction that innovative science translates into therapeutic possibilities for our patients, who are at the heart of everything we do. For more information, please visit .  Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our full year 2024 net product revenue, expectations for the growth and value of WAKIX, plans to submit an sNDA for pitolisant in idiopathic hypersomnia; our future results of operations and financial position, business strategy, products, prospective products, product approvals, the plans and objectives of management for future operations and future results of anticipated products. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: our commercialization efforts and strategy for WAKIX; the rate and degree of market acceptance and clinical utility of pitolisant in additional indications, if approved, and any other product candidates we may develop or acquire, if approved; our research and development plans, including our plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreements with Bioprojet Société Civile de Recherche ("Bioprojet"); the availability of favorable insurance coverage and reimbursement for WAKIX; the timing of, and our ability to obtain, regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and additional financing needs; our ability to identify, acquire and integrate additional products or product candidates with significant commercial potential that are consistent with our commercial objectives; our commercialization, marketing and manufacturing capabilities and strategy; significant competition in our industry; our intellectual property position; loss or retirement of key members of management; failure to successfully execute our growth strategy, including any delays in our planned future growth; our failure to maintain effective internal controls; the impact of government laws and regulations; volatility and fluctuations in the price of our common stock; the significant costs and required management time as a result of operating as a public company; the fact that the price of Harmony's common stock may be volatile and fluctuate substantially; statements related to our intended share repurchases and repurchase timeframe and the significant costs and required management time as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on February 22, 2024, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. Harmony Biosciences Media Contact: Cate McCanless 202-641-6086 cmccanless@harmonybiosciences.com Harmony Biosciences Investor Contact: Luis Sanay, CFA 445-235-8386 lsanay@harmonybiosciences.com Company Codes: NASDAQ-NMS:HRMY

扫描二维码即可下载《数图药讯》2024年第22期目   录政策解读1. 国家医疗保障局：关于发布《药品医保目录归属认定工作程序（试行）》的公告2. 国家药监局：关于适用《Q2（R2）：分析方法验证》《Q14：分析方法开发》国际人用药品注册技术协调会指导原则的公告（2024年第65号）3. 国家药监局药审中心：关于发布《在罕见疾病药物临床研发中应用去中心化临床试验的技术指导原则》的通告（2024年第25号）4. 国家药监局药审中心：第六期“药审云课堂”培训报名通知及下期预告5. 上海阳光医药采购网：关于公示2024年3月部分短缺药全国平均价的公告行业动态1. 药讯动态：重磅获批2. 药讯动态：重磅临床3. 药企动态：市场动态零售品类数据洞见1. 皮肤病药品市场趋势分析2. 复方卡力孜然零售放大市场销售分析3. 白癜风相关治疗药品零售分析政策解读1、国家医疗保障局：关于发布《药品医保目录归属认定工作程序（试行）》的公告为更好保障参保人员基本用药需求，进一步规范医保药品目录管理，提升科学化、精细化管理水平，按照《基本医疗保险用药管理暂行办法》，国家医疗保障局研究制定了《药品医保目录归属认定工作程序（试行）》，现予发布。自发布之日起施行。附件：1.药品医保目录归属认定工作程序（试行）2.药品医保目录归属认定申请表附件详情请扫码2、 国家药监局：关于适用《Q2（R2）：分析方法验证》《Q14：分析方法开发》国际人用药品注册技术协调会指导原则的公告（2024年第65号）为推动药品注册技术标准与国际接轨，经研究，国家药品监督管理局决定适用《Q2（R2）：分析方法验证》《Q14：分析方法开发》国际人用药品注册技术协调会指导原则（以下简称Q2（R2）/Q14）。申请人需在现行药学研究技术要求基础上，按照Q2（R2）/Q14指导原则的要求开展研究；自2024年11月24日开始的相关研究（以试验记录时间点为准），均适用Q2（R2）/Q14指导原则。具体信息请登录国家药监局官网查看。3、国家药监局药审中心：关于发布《在罕见疾病药物临床研发中应用去中心化临床试验的技术指导原则》的通告（2024年第25号）为深化“以患者为中心”的临床试验新理念，助力罕见疾病药物临床研发，在国家药品监督管理局的部署下，药审中心组织制定了《在罕见疾病药物临床研发中应用去中心化临床试验的技术指导原则》。根据《国家药监局综合司关于印发药品技术指导原则发布程序的通知》（药监综药管〔2020〕9号）要求，经国家药品监督管理局审查同意，现予发布，自发布之日起施行。附件：在罕见疾病药物临床研发中应用去中心化临床试验的技术指导原则附件详情请扫码4、国家药监局药审中心：第六期“药审云课堂”培训报名通知及下期预告为进一步促进药品研发，提高注册受理及审评效率，国家药监局药审中心计划于6月7日13:30-17:00在线上直播举办第六期“药审云课堂”，就新发布政策法规、药品审评常见共性问题进行解读。培训面向全社会公开，欢迎研发企业、药品生产企业的注册及研发相关人员参加。具体信息请登录国家药监局药审中心官网查看。附件：1.2024年第6期“药审云课堂”议程.docx2.报名及观看流程.docx相关文件请扫描上方二维码查看5、上海阳光医药采购网：关于公示2024年3月部分短缺药全国平均价的公告根据国家医疗保障局相关文件，为贯彻落实《关于进一步做好短缺药品保供稳价工作的意见》（国办发〔2019〕47号）精神，现公布2024年3月部分短缺药的全国平均价（详见附件），请各医疗机构根据市场供需情况，在保供稳价的基础上，采购质量可靠、价格适宜的药品。附件：2024年3月部分短缺药全国平均价表相关文件请扫描上方二维码查看行业动态01药讯动态：重磅获批和重磅临床最近重磅获批琅钰集团旗下子公司琅铧医药和Bioprojet公司共同申报的盐酸替洛利生片新适应症上市申请获得NMPA批准。替洛利生是⼀种选择性组胺H3受体反向激动剂，本次获批的适应症为：治疗发作性睡病青少年和6岁以上儿童患者的日间过度嗜睡或猝倒。 博安生物度拉糖肽注射液（BA5101）上市申请已获CDE受理，用于成人 2 型糖尿病患者的血糖控制。 深圳华润九新药业有限公司申报的注射用头孢比罗酯钠拟纳入优先审评，适用于治疗成人的下述感染：医院获得性肺炎（HAP），但呼吸机相关性肺炎（VAP）除外；社区获得性肺炎（CAP）。 罗氏Inavolisib在美国的上市申请获得优先审评资格，与哌柏西利和氟维司群联用治疗 PIK3CA 突变、HR+、HER2- 的局部晚期或转移性乳腺癌成人患者。 赛诺菲CD38单抗Sarclisa（艾沙妥昔单抗）联合硼替佐米、来那度胺和地塞米松（VRd）治疗不适合移植、新诊断多发性骨髓瘤患者（NDMM）的新适应症补充申请（sBLA）获得FDA受理并获得优先审评资格。最近重磅临床泽璟制药盐酸吉卡昔替尼片（曾用名：盐酸杰克替尼片）用于治疗12岁及以上青少年和成人非节段型白癜风患者的2/3期临床试验获得中国NMPA批准。 第一三共/阿斯利康在CDE 临床试验登记与信息公示平台登记启动了一项 Dato-DXd（DS-1062a/德达博妥单抗） 新 III 期临床 TROPION-Lung14 研究，联合奥希替尼一线治疗 EGFR 突变阳性局部晚期或转移性 NSCLC 患者（登记号：CTR20241813）。 威尚生物医药新型EGFR抑制剂WSD0922获得FDA批准，在美国开展2期临床试验，用于经一线奥希替尼治疗进展具C797S突变的晚期/转移性非小细胞肺癌。 辐联科技PSMA靶向放射性药物225Ac-FL-020已获得美国FDA新药临床试验（IND）的批准，用于治疗转移性去势抵抗性前列腺癌（mCRPC）。 瑞博生物自主研发的靶向FXI的抗血栓siRNA药物RBD4059获得欧洲药品管理局（EMA）2期临床试验许可。 02药企动态：市场动态5月27日，基石药业宣布与欧洲医药公司Ewopharma达成商业化合作，将舒格利单抗在中东欧地区的商业化权利授予后者。根据许可及商业化协议，Ewopharma将获得舒格利单抗在瑞士以及18个中东欧国家（CEE）的商业化权利。这包括欧盟成员国保加利亚、克罗地亚、捷克共和国、爱沙尼亚、匈牙利、拉脱维亚、立陶宛、波兰、罗马尼亚、斯洛伐克和斯洛文尼亚，以及非欧盟国家阿尔巴尼亚、波斯尼亚和黑塞哥维那、科索沃、北马其顿、摩尔多瓦、黑山和塞尔维亚。基石药业将最高获得5130万美元的首付款，以及后续注册和销售里程碑付款。5月28日，吉利德科学公司(Gilead Sciences）与Cartography Biosciences达成战略合作协议，共同为三阴性乳腺癌（TNBC）和最常见的非小细胞肺癌（NSCLC）腺癌患者发现和开发治疗方法。根据协议条款，Cartography将获得2000万美元的预付款，以及额外的近期临床前里程碑付款。Cartography也有资格获得每个可选项目的开发、监管和商业里程碑付款，以及净销售额的分层版税。零售品类数据洞见01皮肤病药品市场趋势分析图1：2019-2024皮肤病药品零售（放大市场）销售额数据来源： 中国药品零售数据库RPDB药店数据中国医药工业信息中心皮肤病在我国是一种常见病，发病率较高，且有些皮肤病不易根治，治愈后易反复。随着社会的进步，人们所处的生活环境也在不断改变，大气污染日益严重，以及年轻人养成晚睡甚至通宵、高油高脂饮食的习惯，导致皮肤病发病率不断提高。 02复方卡力孜然零售放大市场销售分析图2: 2019-2024年复方卡力孜然零售（放大市场）销售额数据来源： 中国药品零售数据库RPDB药店数据中国医药工业信息中心复方卡力孜然酊，中成药，具有温肤散寒，祛风燥湿，舒经活络，活血化瘀及清除异常粘液质的功效。用于治疗白癜风(白热斯)。 03白癜风相关治疗药品零售分析图3: 2019-2024年白癜风治疗药品零售（放大市场）销售额数据来源： 中国药品零售数据库RPDB药店数据中国医药工业信息中心白癜风是一种原发性的、局限性或泛发性的皮肤黏膜色素脱失症，是临床常见的一种皮肤病，属于自身免疫性疾病。2023年白癜风治疗药品零售（放大市场）销售额占比前三的药品分别为：白灵片（29.86%）、复方卡力孜然酊（16.55%）、白癜风胶囊（12.64%）。来源：医药地理、国家药监局、腾讯、新浪等。END如需获取更多数据洞察信息或公众号内容合作，请联系医药地理小助手微信号：pharmadl001

▎药明康德内容团队报道今日（5月27日），中国国家药监局（NMPA）官网最新公示，由琅钰集团旗下子公司琅铧医药和Bioprojet公司共同申报的盐酸替洛利生片新适应症上市申请获得批准。根据中国国家药监局药品审评中心（CDE）优先审评公示以及公开资料，替洛利生是⼀种选择性组胺H3受体反向激动剂，本次获批的适应症为：治疗发作性睡病青少年和6岁以上儿童患者的日间过度嗜睡或猝倒。截图来源：NMPA官网发作性睡病是一种罕见神经系统疾病，已于2023年9月被纳入中国《第二批罕见病目录》。该疾病常被认为是终身性疾病，多于儿童期（8~10岁）起病，临床上以日间过度嗜睡（EDS）、猝倒（肌肉无力突然发作）、入睡前幻觉、睡眠瘫痪和夜间睡眠紊乱为主要核心症状。发作性睡病严重影响患者的学习/工作能力和身心健康，患者随时面临过度嗜睡和猝倒等带来的生命威胁。公开资料显示，替洛利生（pitolisant）是由Bioprojet公司研发的⼀种选择性组胺H3受体反向激动剂。该药通过一种全新的作用机制发挥作用，即通过增强组胺能神经元活性，增加大脑中促进觉醒的神经递质组胺的合成和释放，进而提高患者的清醒度和警觉性。2020年10月，琅铧医药与Bioprojet公司达成战略合作和独家授权协议，获得替洛利生在中国的独家开发、注册、商业化和生产权益。2023年6月，替洛利生在中国获批用于治疗发作性睡病成人患者的日间过度嗜睡或猝倒。本次替洛利生获批的新适应症针对的是青少年和6岁以上儿童患者。该项上市申请此前也已经被CDE以“符合儿童生理特征的儿童用药品新品种、剂型和规格”为由纳入优先审评。替洛利生治疗6岁及以上发作性睡病儿童患者的3期临床研究积极结果已于2023年4月发表于《柳叶刀-神经病学》（The Lancet Neurology）。研究表明，每天使用5~40mg替洛利生的患者在减少日间过度嗜睡与猝倒方面显示出显著的有效性。替洛利生组和安慰剂组从基线到双盲期结束的发作性睡病量表（UNS）总分平均校正差异分别为-6.3和-2.6，替洛利生组下降幅度更大。此外，替洛利生在患有发作性睡病的儿童和青少年中具有良好的风险收益。（注：UNS用于测量发作性睡病症状的强度和频率，分数越高表示症状越严重）。同时，在安全性方面，替洛利生对儿童的表现与成人一致，不良事件通常较轻微。参考资料：[1] 2024年05月27日药品批准证明文件送达信息. Retrieved May 27, 2024 from https://www.nmpa.gov.cn/zwfw/sdxx/sdxxyp/yppjfb/20240527153320182.html[2]治疗儿童发作性睡病，盐酸替洛利生片被国家药监局纳入优先审评！. Retrieved July 11，2023, from https://mp.weixin.qq.com/s/JAgtNFOPDPGDqaZJ0g6OZg[3] 柳叶刀：替洛利生在发作性睡病儿童患者的III期临床研究中结果积极. Retrieved Apr 13，2023, from https://mp.weixin.qq.com/s/8JcLjOWazULQvBWg8J3NDA本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。