FoldRx Pharmaceuticals LLC

– Dr. Laura Bessen Brings More Than Two Decades of Experience Across Medical Affairs and Clinical Development in Support of Successful Product Launches – – Dr. Donna Grogan Joins as Accomplished Drug Development and Regulatory Strategy Leader With More than 25 Years of Experience in Developing Novel Therapeutics – – Appointments Highlight C4T’s Continued Commitment to Evolving Board Composition – WATERTOWN, Mass., Aug. 02, 2022 (GLOBE NEWSWIRE) -- C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, today announced the appointments of Laura Bessen, M.D. and Donna Grogan, M.D. to its board of directors. “We are delighted to announce Laura and Donna as C4T’s newest directors after an extensive search that included a talented and diverse pool of clinical development leaders experienced in helping companies bring innovative therapies to patients,” said Bruce Downey, chairman of C4 Therapeutics. “On behalf of the board of directors, I look forward to working closely with Laura and Donna as C4T continues to evolve in our pursuit of delivering on the potential of targeted protein degradation science to create a new generation of medicines and change how diseases are treated.” “I am eager to use my broad experiences in medical affairs and clinical development to help C4T advance multiple oncology programs to benefit patients,” said Laura Bessen, M.D. “C4T is at a transformational stage, with two programs in the clinic and additional programs preparing to advance into clinical trials. I am excited to help C4T capitalize upon opportunities ahead.” Commenting on joining the C4T board of directors, Donna Grogan, M.D. said, “It is an exciting time to join C4T and partner with the company to navigate drug development and regulatory milestones to advance an innovative portfolio of small-molecule medicines. I look forward to working with my C4T colleagues to help the Company apply its industry-leading targeted protein degradation science to transform how patients are treated.” About Laura Bessen, M.D.Laura Bessen, M.D. currently serves as managing partner at Maxsam Advisors LLC where she provides strategic clinical and medical affairs advice to clients across the biotechnology and pharmaceutical industries. Previously, she held roles of increasing responsibility over a 15-year career at Bristol Myers Squibb (BMS), most recently as Vice President, Head of US Medical. During her tenure at BMS, she helped launch 11 new products including Opdivo®, Yervoy®, elotuzomab, and Eliquis® and co-led BMS’ partnership with Gilead to develop Atripla®. Dr. Bessen also co-led the development of BMS product commercialization model, life cycle management and launch investment principles. Earlier in her career, Dr. Bessen served in medical affairs roles at DuPont Pharmaceuticals. She currently serves on the Board of Directors of Artiva Biotherapeutics, an oncology company developing and advancing off-the-shelf, allogeneic natural killer cell therapies for patients with hematologic cancers or solid tumors. Dr. Bessen received her M.D. degree from New York University School of Medicine and B.S. in biochemistry from the State University of New York at Binghamton. About Donna Grogan, M.D.Donna Grogan, M.D. currently serves as Principal of Grogan Consulting LLC where she supports clients across drug development, regulatory strategy, trial design and data interpretation. Between September 2013 and June 2019, Dr. Grogan served as Chief Medical Officer of Clementia Pharmaceuticals, which was acquired by Ipsen in April 2019. She previously served as Chief Medical Officer for several HealthCare Ventures portfolio companies including Anexon, Apofore, and DecImmune. Between February 2007 and August 2011, Dr. Grogan served as Chief Medical Officer, Senior Vice President Clinical Development at FoldRx Pharmaceuticals, which was acquired by Pfizer in October 2010. Earlier in her career, she held roles of increasing responsibility at Sepracor, Inc. where she was involved in multiple high-profile product approvals including Lunesta®, Xopenex HFA®, and Brovana™. Dr. Grogan previously served as a board member, including membership on the Compensation Committee and the Scientific Committee, of Momenta Pharmaceuticals until it was acquired by J&J in October 2020. She holds a M.D. from University of Illinois College of Medicine and a B.A. from College of the Holy Cross. About C4 TherapeuticsC4 Therapeutics (C4T) (Nasdaq: CCCC) is a clinical-stage biopharmaceutical company dedicated to delivering on the promise of targeted protein degradation science to create a new generation of medicines that transform patients’ lives. C4T is leveraging its TORPEDO® platform to efficiently design and optimize small-molecule medicines that harness the body’s natural protein recycling system to rapidly degrade disease-causing proteins, offering the potential to overcome drug resistance, drug undruggable targets and improve patient outcomes. C4T is advancing multiple targeted oncology programs to the clinic and expanding its research platform to deliver the next wave of medicines for difficult-to-treat diseases. For more information, please visit www.c4therapeutics.com. Forward-Looking StatementsThis press release contains “forward-looking statements” of C4 Therapeutics, Inc. within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements may include, but may not be limited to, express or implied statements regarding our ability to develop potential therapies for patients; the design and potential efficacy of our therapeutic approaches; the predictive capability of our TORPEDO® platform in the development of novel, selective, orally bioavailable degraders; the potential timing, design and advancement of our pre-clinical studies and clinical trials, including the potential timing for regulatory authorization related to clinical trials and other clinical development activities; our ability and the potential to successfully manufacture and supply our product candidates for clinical trials; our ability to replicate results achieved in our pre-clinical studies or clinical trials in any future studies or trials; and regulatory developments in the United States and foreign countries. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties related to the initiation, timing, advancement and conduct of pre-clinical and clinical studies and other development requirements for our product candidates; the risk that any one or more of our product candidates will cost more to develop or may not be successfully developed and commercialized; and the risk that the results of pre-clinical studies and/or clinical trials will or will not be predictive of results in connection with future studies or trials. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in C4 Therapeutics’ most recent Annual Report on Form 10-K and/or Quarterly Report on Form 10-Q, as filed with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and C4 Therapeutics undertakes no duty to update this information unless required by law. Investor Contact: Kendra Adams SVP, Communications & Investor Relations Kendra.Adams@c4therapeutics.com Media Contact: Loraine Spreen Director, Corporate Communications & Patient Advocacy LSpreen@c4therapeutics.com Photos accompanying this announcement are available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/99a1b2ca-915c-446c-95e3-28fb99720bcb https://www.globenewswire.com/NewsRoom/AttachmentNg/4480470b-bbb5-4eb9-bc72-241940f28d18

Philippe Dro, Luciole Medical, and Kush Parmar, 5AM Ventures, named to Board of Directors SCHLIEREN, Switzerland and BOSTON, July 12, 2022 /PRNewswire/ -- GlycoEra AG announced today key appointments of biotech industry leaders to its Board of Directors, including Georges Gemayel, Ph.D., as Chair, as well as Philippe Dro, Ph.D., M.B.A., and Kush Parmar, M.D., Ph.D. Dr. Gemayel is an industry veteran with more than 30 years' experience including global management, executive positions and a range of board appointments. Dr. Dro is Chairman of the Board and Chief Executive Officer at Luciole Medical and has deep experience in biotech business strategy at various emerging biotechnology and medtech companies. Dr. Parmar is a physician-scientist and Managing Partner at 5AM Ventures, where he is focused on building next-generation life science companies. "I am thrilled to welcome Georges, Philippe, and Kush to the GlycoEra Board of Directors as GlycoEra continues to grow and assemble a world-class team to realize the full potential of our CustomGlycan glycoengineering platform," said Ganesh V. Kaundinya, Ph.D., President and Chief Executive Officer of GlycoEra. "Georges' international biopharmaceutical leadership experience and track record of developing successful medicines will prove invaluable as we build a high-value pipeline of medicines. In addition, both Philippe and Kush bring strategic, business and company building expertise to help guide our trajectory of growth." Dr. Gemayel served as President and CEO of Altus Pharmaceuticals, Executive Vice President at Genzyme Corporation, and Vice President, National Specialty Care at Roche Labs Inc. Dr. Gemayel is currently the Chair of the Boards of Dynacure, Gemini Therapeutics, and Enterome SA. Previously, Dr. Gemayel served as Executive Chair of FoldRx Pharmaceuticals and of Syndexa Pharmaceuticals, as Chair of Dimension Therapeutics, OxThera, Orphazyme A/S and Epitherapeutics, and as Director of Prosensa, Raptor Pharmaceuticals, NPS Pharma, Momenta Pharmaceuticals, and Adolor. Dr. Gemayel completed his doctorate in pharmacy at St. Joseph University in Beirut, Lebanon and earned a Ph.D. in pharmacology at Paris-Sud University in Paris, France. "I am impressed with the power of the CustomGlycan Platform to create a portfolio of novel biological products that degrade circulating and membrane proteins in a highly selective and targeted fashion. Removing and degrading disease-causing proteins has broad potential therapeutic applications across autoimmune diseases, oncology, neurology, allergy, and infectious diseases," said Dr. Gemayel. "I am excited to collaborate with the experienced leadership team and Board, including Philippe and Kush, to deliver on our shared vision of developing novel biologics for a wide range of indications." In addition to Dr. Gemayel, Drs. Dro and Parmar bring years of experience and deep knowledge in biotech leadership to GlycoEra: Dr. Philippe Dro, Chairman of the Board and Chief Executive Officer, Luciole Medical Dr. Dro has deep experience initiating and executing strategic transactions at various emerging biotechnology and medtech companies. He is currently Chairman of the Board and CEO at Luciole Medical, while also serving on the Board of several life science companies. He was CEO and member of the Supervisory Board at GlycoVaxyn AG prior to its acquisition by GlaxoSmithKline. Previously, Dr. Dro was CEO and Chairman of the Board of Endoart SA, where he led the company through a successful turnaround resulting in its acquisition by Allergan. He also was a member of the founding team at Axovan, a biotechnology company acquired by Actelion in 2003. Prior to that, he served in management positions at Novartis, SkyePharma, and Antares Pharma. Dr. Kush Parmar, Managing Partner, 5AM Ventures Dr. Parmar currently serves as a Director on the Boards of both private and public companies, including Akouos, Ensoma, Entrada Therapeutics, Homology Medicines, Rallybio, and Vor Biopharma. He previously served as Board Member or Observer for Achaogen, Arvinas, Audentes, Envoy (acquired by Takeda), and scPharmaceuticals, and as Acting VP of Strategy and Corporate Development at Novira (acquired by Johnson & Johnson). Dr. Parmar serves on the Advisory Boards of Penn Medicine, Princeton University's Department of Molecular Biology, and the Grace Science Foundation, and is a fellow of the Society of Kauffman Fellows. Drs. Gemayel, Dro, and Parmar join Monique Schiersing of Roche Venture Fund, Graziano Seghezzi of Sofinnova Partners, and Dr. Kaundinya on the Board of Directors. About GlycoEra AG GlycoEra AG is a biotechnology company building a high-value pipeline of medicines based on deep expertise in glycobiology and protein degradation. GlycoEra's innovative CustomGlycan platform enables the design, engineering, and development of novel biologics for a broad range of indications. GlycoEra is advancing a range of assets toward human clinical studies. The company raised more than $49 million (CHF 45 million) in a 2021 Series A financing round co-led by 5AM Ventures, Sofinnova Partners, and Roche Venture fund. GlycoEra is based in Schlieren, Switzerland and Boston, Mass., USA. For further information, please visit .

2019年上市的药品中，2021年销售额超10亿美元的有3款药，其中2款为罕见病药——Trikafta、Vyndaqel/Vyndamax；2020年上市的药品中，2021年销售额超5亿美元的有3款药，其中2款为罕见病药——Tepezza 、Evrysdi。Trikafta、Vyndaqel/Vyndamax和Tepezza的2021年销售额已超10亿美元，分别为57亿美元、20.15亿美元和16.6亿美元。这3款成功的罕见病药带给我们什么启示？相关流行病学及竞争格局先来了解3个药品涉及的流行病学及它们的竞争格局。Trikafta（福泰制药）、Vyndaqel/Vyndamax（辉瑞）和Tepezza（Horizon）获批的适应症分别为：“6岁及以上CFTR 基因中至少有一个 F508del 突变或基于体外数据有反应的 CFTR 基因突变的囊性纤维化患者（CF）”、“成人野生型或遗传性转甲状腺素蛋白淀粉样变性的心肌病（ATTR-CM）”和“甲状腺眼病（TED）”。（注：Vyndaqel和Vyndamax是tafamidis的两种制剂，1粒61mg Vyndamax胶囊等效于4粒20mg Vyndaqel胶囊）CFCF是一种罕见的、缩短寿命的遗传疾病，由CFTR基因突变引起。该基因中大约有2000个已确定的突变，其中127个目前已知会导致CF。北美、欧洲和澳大利亚有7.5万人患有CF，全球约10万人患有CF。美国每年新增约1000例CF患者（来源：福泰制药官网）。据mayoclinic ，自2012年以来，CF只有靶向CFTR基因突变这一种有效“对因”疗法，其他药物为“对症”疗法。靶向CFTR基因突变的药物经历从单药（Ivacaftor）到2联（lumacaftor/ivacaftor或tezacaftor/ivacaftor）再到3联疗法（elexacaftor/ivacaftor/tezacaftor）的迭代。Trikafta是靶向CFTR基因突变的3联疗法，竞品只有同公司的单药和2联疗法。单药（Kalydeco）于2012年推出，适用于约4-5%的CF患者，2联疗法Orkambi（lumacaftor / ivacaftor）和Symdeko / Symkevi（tezaftor / ivacaftor）分别在2015年和2018年将覆盖范围扩展到CF人群的一半左右，Trikafta可以面向90%的CF患者。图1 CF治疗药物竞争格局（来源：mayoclinic，丰硕创投整理）ATTR-CM据估计，全球约有ATTR-CM患者40万，美国约有10万，其中只有1%至2%的患者被诊断出来。通常，ATTR-CM仅在症状变得严重后才被诊断出来。一旦确诊，ATTR-CM 患者的中位预期寿命约为 2 至 3.5 年（来源：辉瑞官网）。目前为止，Vyndaqel/Vyndamax是唯一一个获批治疗ATTR-CM的药物。TEDTED是一种严重的，进行性的和威胁视力的罕见自身免疫性疾病。TED的确切患病率尚不清楚，但估计一般人群中每10万名女性中有16人，每10万名男性中有2.9人，即TED影响全球数十万人（来源：rarediseases.org）。目前为止，Tepezza是唯一一个获批用于治疗TED的药物。此前，轻度患者可用人工泪液缓解，中重度患者（占所有TED患者的1/3）可用用皮质类固醇、环孢素等免疫抑制和放疗等疗法，但是类固醇在减少眼球突出方面不是很有效，且约1/3接受以上疗法的患者会复发（来源：和铂医药招股书）。小结：这3个畅销药物面向的患者群不算十分小（全球10万~数十万）。Vyndaqel/Vyndamax和Tepezza分别是唯一一个获批用于治疗ATTR-CM和TED的药物。Trikafta是 CF唯一一类“对因疗法”—— 靶向CFTR基因突变的最新一代产品，竞品是同一家公司（福泰制药）的老产品。对投资的启示（1）什么样的管线更有投资价值讨论3个药品背后的流行病学、竞争格局、开发阶段、原研背景。流行病学、竞争格局3个药物的流行病学和竞争格局告诉我们，这类罕见病药物有比较大的想象空间：（1）没有竞争对手，目标患者人群在10万以上；（2）唯一一类治疗药物的最新一代产品，目标患者人群在10万以上；（3）疾病诊断率较低，但目标患者人群达到40万+，且没有竞争对手。开发阶段一般哪个阶段的结果更能确定管线的价值呢？看公司股价表现或管线被收购时所处的开发阶段。Trikafta从发现到上市都在福泰制药进行，直接看福泰制药的股价表现。当Ivacaftor的2期结果披露时，福泰制药的股价变动不大（25美元左右）；披露Ivacaftor的3期结果时，股价已经来到40美元左右；披露 Ivacaftor/lumacaftor 二联的2期结果结果时，股价上到60美元左右；Ivacaftor/lumacaftor 二联的3期达到主要终点，股价上到95美元左右；Ivacaftor/lumacaftor 二联获批时，股价上到130美元左右；三联疗法的1和2期结果出来时，股价变动不大；启动elexacaftor/Tezacaftor/Ivacaftor的三联疗法时，股价去到160美元；三联疗法达到主要终点时，股价去到180美元；三联疗法获批时股价变动不大；2019年年报出来，股价去到230美元（三联疗法销售超预期）；2020年一季报出来，股价去到250美元（三联疗法销售再超预期）；截止2022年6月1日，福泰制药的收盘价为270美元。图2 福泰制药收盘价及药物研发阶段（来源：福泰制药官网、choice，丰硕创投整理）Vyndaqel的原研是FoldRx，一个非上市公司。在2010年辉瑞收购了FoldRx，当时Tafamidis治疗ATTR-PN的开发已经进入到上市申请阶段， 治疗的ATTR-CM的开发处于II期临床阶段。Tepezza的原研是罗氏，River Vision Development Corporation（RVDC）于2012年获得TEPEZZA的许可，开始治疗眼科疾病的研究。Horizon于2017年收购RVDC，收购时，River Vision刚公布Tepezza治疗TED的II期积极结果不久。综上，罕见病管线的价值一般在II期概念性验证成功之后得到认可。原研背景罕见病药物开发风险很高，什么原研背景相对值得信赖呢？以福泰制药为例，它对CF药物的研发背靠诺奖得主钱永健创立Aurora高通量药物分子筛选平台，还有CF基金会的坚定支持，可谓既有技术又有稳定的资金支持，它的成功存在一定的必然性。以Vyndaqel的原研FoldRx为例，在发现Vyndaqel以前，FoldRx的创始人Kelly已经在实验室对抑制转甲状腺素蛋白原纤维形成的方法研究了10几年，直到发现了有效成分Tafamidis之后才创立的FoldRx，十几年对一类疾病的研究是Vyndaqel成功的底气。TEPEZZA呢，发现分子的是大厂罗氏，第一个研究它在TED中潜力的公司是River Vision。River Vision由投资管理公司Narrow River Management创立，公司的CEO David M. Madden也是Narrow River Management的创始人兼负责人。此前，Madden曾担任生物制药公司Adolor Corporation（NASDAQ:ADLR）的临时总裁兼CEO，因研发出有吸引力的药物管线，该公司后来被 Cubist Pharmaceuticals（NASDAQ:CBST）收购。大厂发现，在曾领导过因成功研发药物而被收购公司的创始人底下进行早期TED试验，并获得不错的结果，这些为TEPEZZA的成功增添了不少信心。小结：当你发现一个罕见病管线的流行病学和竞争格局如上述般理想，II期概念性验证也成功了，还有可靠的原研背景加持，那应该是个不错的投资机会。（2）估值时，每年渗透率给多少结合这三个药物的季度销售额、环比增长率、获批适应症、获批地区、获批地区目标患者人数和年费来分析。Trikafta截止2021年6月，Trikafta已经在美国、瑞士、澳大利亚和以色列以及欧盟和英国获批。自上市以来，Trikafta的季度销售额及环比增长率如下图所示。图3 Trikafta季度销售额及环比增长率（来源：福泰制药财报，丰硕创投整理）据SEC，Trikafta年费约为31万美元，那么2020年、2021年，Trikafta约分别覆盖了1.2万和1.8万CF患者，即Trikafta上市第二年大约覆盖了获批地区12%的CF患者，第三年则为18%。Vyndaqel/VyndamaxVyndaqel于2011年11月获欧洲药品管理局批准，用于治疗ATTR-PN。2018年，Vyndaqel销售额为1.48亿美元，一直到2019年获批ATTR-CM适应症以前，Vyndaqel的销售额不温不火。2019年3月、5月以及2020年2月分别在日本、美国、欧洲推出ATTR-CM适应症后，Vyndaqel/Vyndamax的销售额迅速放量（2019 Q2环比增长53.66%，2019 Q3环比增长147.62%，2020Q2环比增长19.91%）。截止2021年12月，Vyndaqel/Vyndamax被批准用于治疗超过55个国家的ATTR-CM，包括美国，欧盟，中国、巴西，阿联酋和加拿大等。自2018年Q3以来，Vyndaqel/Vyndamax的季度销售额及环比增长率如下图所示。图4 Vyndaqel/Vyndamax季度销售额及环比增长率（来源：辉瑞财报，丰硕创投整理）据fiercepharma，Vyndaqel/Vyndamax的年费约为22.5万美元。假定ATTR-PN适应症的销售额以每个季度增长5%（与2018Q4、2019Q1的增速接近）的速度增加，那么用Vyndaqel/Vyndamax的总销售额扣除ATTR-PN适应症的销售额，推算ATTR-CM销售额。经计算，2019年、2020年、2021年，Trikafta约分别覆盖了1300、4800、7800个ATTR-CM患者，覆盖率约为0.3%、1.2%、2.0%。该覆盖率比较低可能是因为ATTR-CM的诊断率比较低。在Vyndaqel推出之前，只有大约1000至2000名ATTR-CM患者得到正确诊断。现在，随着辉瑞公司疾病意识的推动和对特定心脏成像技术的更好采用，辉瑞公司的估计诊断率已上升至13%（来源：辉瑞2020年4月下旬电话会议）。TEPEZZATEPEZZA目前仅在美国获得批准。自上市以来，TEPEZZA的季度销售额及环比增长率如下图所示。2021Q1 TEPEZZA的销售额暴跌至210万美元，是因为美国政府强制Horizon的制造合作伙伴实施的COVID 19疫苗订单，导致的短期TEPEZZA供应中断。供应恢复后，TEPEZZA销售由恢复了增长。据Jefferies的报道，一个患者接受6个月疗程的TEPEZZA治疗，平均需要花费35万美元。那么2020年、2021年，TEPEZZA约分别覆盖了个2300、4700个TED患者。美国大概有3万TED患者，1万中重度TED患者，TEPEZZA面向中重度TED患者。即TEPEZZA上市第一年对美国目标TED患者的覆盖率约达23%，第二年约达47%。图5 TEPEZZA季度销售额及环比增长率（来源：Horizon财报，丰硕创投整理）小结：对于ATTR-CM、TED这些还没有标准疗法的适应症，及CF这种已经有且只有一类标准疗法的适应症，即将获批药物若为唯一标准疗法或最新一代标准疗法，且疗效明显，安全性良好，那么它们上市会快速获得不错的销售额。像TEPEZZA这种没有竞争对手的，渗透得最快，上市第一、二年渗透率能去到23%和47%。像Trikafta这种是唯一一类治疗药物的最新一代产品，竞争对手是旧两代产品，渗透得也相对快，上市第二、三年的渗透率去到12%和18%。像Vyndaqel/Vyndamax这种没有竞争对手，但是对应疾病诊断率很低，渗透率要给低一些，Vyndaqel/Vyndamax头三年渗透率只有0.3-2.0%。对罕见病药物开发的启示分工启示通常是有经验的小药企打头阵，然后在研发的某个阶段（通常是II期验证成功后）交到大药企手里快速推进后期临床乃至商业化。获得研发资金的启示可以向福泰制药学习，立项之前寻找CF基金会这样坚定的合作伙伴，这样可以减少因为资金不足而前功尽弃的顾虑。福泰制药研发模式的启示1989年，徐立之和弗朗西斯·柯林斯（Francis S. Collins, 现美国国立卫生研究院/NIH院长）共同领导的团队首次发现了CF的致病基因。Aurora进一步发现，CF病人的蛋白缺陷可以大体分为两种情况，第一种情况是在病人上皮细胞膜的表面有足够多的CFTR蛋白，但该蛋白无法产生足够大的通道开口确保离子的正常进出；另外一种情况则是病人自身无法产生足够量的CFTR蛋白到达细胞膜表面。在新的经过验证的靶点出现以前，福泰制药针对CFTR基因突变的两种机制，及“病人自身无法产生足够量的CFTR蛋白的情况更常见”的进一步发现，不断推出新的疗法，将使用患者范围从4-5%，扩展到90%。Vertex的首席战略官David Altshuler表示“如果你发现了一种药物，你做对了一切，但目标是错误的，它永远不会成功。”。专注于经过验证的药物靶点，对罕见病研发不失为一种好策略。对中国药企的启示也许罕见病药物并不像我们想象中的“低投资回报”。以Vyndamax为例，它在美国的价格是20622美元/瓶（61mgX30粒），同样的规格，2021年纳入中国医保后，价格为24650人民币，年费大概是在27万。所以理想的罕见病药物在中国也能在2-3年内达到超过10亿人民币的销售额。同时，罕见病药物因为可选择性少，还可以节省不少营销费用。另外，近年来，国家出台了不少罕见病药物支持政策。中国药企可以通过自主研发，或者通引进国外原研背景较好的早期管线，研发罕见病药物，提高罕见病药品的可及性。声明：以上内容仅供参考，不构成投资建议。