Leadiant Biosciences, Inc.

DUBLIN, Oct. 2, 2023 /PRNewswire/ -- The "Enzyme Replacement Therapy Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering. The global enzyme replacement therapy market is expected to grow from $10.03 billion in 2022 to $10.85 billion in 2023 at a compound annual growth rate (CAGR) of 8.1%. The market is expected to reach $14.79 billion in 2027 at a CAGR of 8.1%. In the forefront of medical advancement, the enzyme replacement therapy (ERT) market stands tall, offering vital treatment avenues for individuals grappling with chronic disorders arising from enzyme deficiencies. The research report delves deep into the nuances of the ERT sector, highlighting market statistics, regional shares, and key players commanding significant market share. ERT's pivotal role in normalizing urinary GAG levels and promoting liver and spleen health is becoming increasingly recognized. Primary enzymes like agalsidase alfa - a critical tool in the combat against Fabry's disease - are reshaping the therapeutic landscape. Our comprehensive report also categorizes other prominent enzymes used, shedding light on their applications in conditions such as Gaucher disease, Pompe disease, and more. A spotlight trend in the ERT realm is the surge in product approvals. For instance, the recent FDA nod to Lamzede (velmanase alfa) in February 2023 marked a significant milestone. As the inaugural enzyme replacement therapy greenlit in the U.S for tackling the non-central neurological manifestations of alpha-mannosidosis, Lamzede offers a promising beacon of hope. Moreover, with the escalating prevalence of rare, chronic, and genetic disorders, the ERT market trajectory looks set for robust growth. The U.S National Library of Medicine projects a near-doubling of individuals aged 50 and above with a chronic ailment by 2050, further emphasizing the critical need for effective enzyme replacement therapies. The report also touches upon the market revenues, detailing contributions from key treatments like Sebelipase alfa, Elosulfase alfa, and more. Our exhaustive coverage ensures businesses and stakeholders are well-armed with insights to navigate the dynamic ERT ecosystem. In a geographical context, while North America reigned supreme in the ERT domain in 2022, the forecasted period paints a promising picture for the Asia-Pacific region. Our study covers a broad spectrum of nations from Australia to the USA, offering a global perspective on this burgeoning market. Harness the power of data-driven insights, industry trends, and strategic opportunities with our comprehensive report, and be poised at the cusp of ERT innovations and advancements. Major players in the enzyme replacement therapy market are Biomarin Pharmaceutical Inc. Leadiant Biosciences Inc. Pfizer Inc. Sanofi S.A. AbbVie Inc. Takeda Pharmaceutical Company Limited Protalix BioTherapeutics Inc. Amicus Therapeutics Inc. Johnson & Johnson Services Inc. Digestive Care Inc. Nestle Health Science Alexion Pharmaceuticals Inc. Allergan Plc. Actelion Pharmaceuticals Ltd. CANbridge Life Sciences Ltd. Chiesi Farmaceutici S.P.A. Astrazeneca Plc. Genzyme Corporation JCR Pharmaceutical Co. Ltd. TEIJIN LIMITED Horizon Therapeutics Plc. Perseo Pharma AG SmartPharm Therapeutics Inc. Key Topics Covered: 1. Executive Summary 2. Enzyme Replacement Therapy Market Characteristics 3. Enzyme Replacement Therapy Market Trends And Strategies 4. Enzyme Replacement Therapy Market - Macro Economic Scenario 4.1. COVID-19 Impact On Enzyme Replacement Therapy Market 4.2. Ukraine-Russia War Impact On Enzyme Replacement Therapy Market 4.3. Impact Of High Inflation On Enzyme Replacement Therapy Market 5. Enzyme Replacement Therapy Market Size And Growth 5.1. Global Enzyme Replacement Therapy Historic Market, 2017-2022, $ Billion 5.1.1. Drivers Of The Market 5.1.2. Restraints On The Market 5.2. Global Enzyme Replacement Therapy Forecast Market, 2022-2027F, 2032F, $ Billion 5.2.1. Drivers Of The Market 5.2.2. Restraints On the Market 6. Enzyme Replacement Therapy Market Segmentation 6.1. Global Enzyme Replacement Therapy Market, Segmentation By Enzyme Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Agalsidase Alfa Agalsidase Beta Galsulfase Other Enzyme Types 6.2. Global Enzyme Replacement Therapy Market, Segmentation By Application, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Gaucher Disease Pompe Disease Fabry Disease Other Applications 6.3. Global Enzyme Replacement Therapy Market, Segmentation By Route Of Administration, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Oral Parenteral 6.4. Global Enzyme Replacement Therapy Market, Segmentation By End-Users, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Hospitals Infusion Centers Other End-Users 7. Enzyme Replacement Therapy Market Regional And Country Analysis 7.1. Global Enzyme Replacement Therapy Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion 7.2. Global Enzyme Replacement Therapy Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1904 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

LA JOLLA, Calif., May 23, 2023 (GLOBE NEWSWIRE) -- CalciMedica Inc. (“CalciMedica”) (OTCQB: CALC), a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening inflammatory diseases with high unmet need, today announced the appointments of Raven Jaeger, M.S., as Chief Regulatory Officer and Andrew Cunningham, M.D., MRCPI, as Senior Vice President of Clinical Development. Both Mrs. Jaeger and Dr. Cunningham bring extensive expertise to their positions on the CalciMedica team. Mrs. Jaeger has over 20 years of experience in the pharmaceutical and biotech industries as a Regulatory Affairs professional in both investigative and commercialized drugs and biologics across a variety of therapeutic areas. Most recently, she served as the Chief Regulatory Officer at Diffusion Pharmaceuticals, Inc. where she supported the product development program and various business initiatives. Prior to this, Mrs. Jaeger held Regulatory Affairs positions at other leading pharmaceutical companies including BridgeBio Pharma, Leadiant BioSciences, Inc. and Nabi Biopharmaceuticals. While at BridgeBio Pharma, she led her team to the first FDA approved New Drug Application across all subsidiaries of the organization. “I am excited to join the CalciMedica team and to build on the clinical progress of Auxora in multiple indications by spearheading regulatory strategy,” said Mrs. Jaeger. “Throughout my career, I have been fortunate to play important roles in the advancement of multiple therapies through the clinic and to regulatory approval, and I look forward to applying my knowledge and experience to the CalciMedica pipeline.” Dr. Cunningham has over 10 years of clinical development experience in the pharmaceutical industry. Prior to this position, Dr. Cunningham served as a consultant for CalciMedica since December 2022. He has valuable leadership and clinical development experience, having served as Head of the Neurodegenerative Franchise at Taysha Gene Therapies and Associate Vice President of Clinical Development at Reata Pharmaceuticals. In his prior Medical Director positions at Takeda Pharmaceutical Company, Sanofi Genzyme and Reata Pharmaceuticals, Dr. Cunningham helped advance drug candidates through all stages of clinical development as well as participated in the filing of New Drug Applications. “I was impressed with Auxora’s potential to treat inflammatory diseases which currently have no approved therapies,” said Dr. Cunningham. “I look forward to applying my skills and experience in clinical development as we continue progress in the Phase 2b CARPO trial for acute pancreatitis and advance our pipeline by expanding the Phase 1/2 CRSPA trial in asparaginase-associated pancreatitis, further investigating Auxora in acute kidney injury and exploring new oral agents for chronic pancreatitis and other chronic inflammatory indications.” “Following a successful merger, these appointments underscore CalciMedica’s commitment to grow and expand our clinical programs. We are delighted to welcome Raven and Andrew to the CalciMedica team,” said Rachel Leheny, Ph.D., Chief Executive Officer of CalciMedica. “In this new phase as a public company, CalciMedica is equipped to advance our mission to develop therapies for critically ill patients whose needs are unmet by the current therapies.” About CalciMedica CalciMedica is a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening inflammatory diseases with high unmet need. CalciMedica’s proprietary technology targets the inhibition of CRAC channels designed to modulate the immune response and protect against tissue cell injury, with the potential to provide therapeutic benefits in life-threatening inflammatory diseases for which there are currently no approved therapies. CalciMedica’s lead product candidate Auxora, a proprietary, intravenous-formulated CRAC channel inhibitor, has demonstrated positive and consistent clinical results in four completed efficacy clinical trials. Auxora is in development for acute pancreatitis with systemic inflammatory response syndrome and asparaginase-associated pancreatitis. CalciMedica was founded by scientists from Torrey Pines Therapeutics and the Harvard CBR Institute for Biomedical Research, and is headquartered in La Jolla, CA. For more information, please visit . Forward-Looking Statements This communication contains forward-looking statements which include, but are not limited to, statements regarding the design and potential benefits of Auxora; CalciMedica’s ongoing and planned clinical trials; and the development and outcomes of CARPO and CRSPA trial programs. These forward-looking statements are subject to the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. CalciMedica’s expectations and beliefs regarding these matters may not materialize. Actual outcomes and results may differ materially from those contemplated by these forward-looking statements as a result of uncertainties, risks, and changes in circumstances, including but not limited to risks and uncertainties related to: the impact of fluctuations in global financial markets on CalciMedica’s business and the actions it may take in response thereto; CalciMedica’s ability to execute its plans and strategies; the ability to obtain and maintain regulatory approval for Auxora; results from clinical trials may not be indicative of results that may be observed in the future; potential safety and other complications from Auxora; the scope progress and expansion of developing and commercializing Auxora; the size and growth of the market therefor and the rate and degree of market acceptance thereof; economic, business, competitive, and/or regulatory factors affecting the business of CalciMedica generally; CalciMedica’s ability to protect its intellectual property position; and the impact of government laws and regulations. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption “Risk Factors” in CalciMedica’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2023 and elsewhere in CalciMedica’s subsequent reports on Form 10-K, Form 10-Q or Form 8-K filed with the SEC from time to time and available at These documents can be accessed on CalciMedica’s web page at ir.calcimedica.com/financials-filings/sec-filings. CalciMedica Contact: Investors and Media Argot Partners Sarah Sutton/Kevin Murphy calcimedica@argotpartners.com (212) 600-1902

近日（2022年11月15日），Leadiant公司被西班牙监管机构罚款1030万欧元(1060万美元)，原因是该制药公司滥用其制造和供应一种治疗脑腱黄瘤病（CTX）罕见病药物鹅去氧胆酸(CDCA）的市场支配地位。经该监管机构调查发现，过去几年来，CDCA的定价涨幅异常之高。2010年，CDCA在西班牙的定价是该地区销售另一种几乎相同药物的14倍。CTX是一种极其罕见的遗传性代谢疾病，由导致CDCA胆汁酸合成的一种酶的缺陷引起，表现为全身症状（儿童慢性腹泻、青少年白内障和肌腱黄色瘤）和神经系统症状（认知能力下降）。几十年来，XCT一直使用以CDCA作为活性成分的药物进行治疗，CDCA的功效已被证明可以预防疾病的进展，甚至可以部分逆转症状。CDCA最早是一种治疗胆结石的药，之后被发现在治疗CTX上疗效显著后，便被临床医生以CTX的超适应症使用。自2010年以来，Leadiant一直是CDCA唯一供应商。2017年，CDCA被获得批准上市，用于CTX的治疗。为了实现市场独占权，Leadiant进行了一系列措施，包括签订排他性条款，打击竞品。该司此前与唯一被授权供应CDCA活性成分的供应商签订了一份排他性条款，阻止其他替代药物的出现。事实上，此前阿姆斯特丹大学医学中心（UMC）也因为生产了一种CDCA替代药而遭到了Leadiant的投诉，直到2020年才得以重新生产。价格上涨超1000倍此次监管部门的调查源于“消费者与用户组织”对该药品超高涨价的投诉，来自两名投诉人均表示，其价格比2008年之前高出1000多倍。2010年9月-2017年6月间，CDCA的价格从984欧元/包上涨到了14618欧元/包。此前，因为该公司的过高定价，意大利和荷兰的监管机构也对该公司提起了诉讼。去年9月，Leadiant已被荷兰消费者和市场管理局进行了1950万欧元的罚款，这也是该机构首次因过高定价对制药公司采取罚款处罚。根据西班牙监管机构此次处罚警告，除了接受罚款之外，Leadiant还必须解除对该产品有效成分的过度控制，并按与卫生部协商的价格后，再进行销售。参考出处：1.https://endpts.com/drugmaker-fined-by-spanish-regulators-10m-for-excessive-price-of-rare-disease-drug/2.案例分析：因过高定价，荷兰对制药公司处以1,950万欧元罚款/泰乐信律师事务所3.让人买不起：一剂药卖1.4万欧元，制药公司被政府罚款1025万欧元！/华人分类信息网4.荷兰监管机构因罕见病药物定价过高对制药商处以 2300 万美元罚款/识林5.荷兰医生抗议药企提价，法国政府施压“救命药”降价/欧时大参近期热门视频更多精彩视频，尽在佰傲谷视频号，欢迎关注~本周好文推荐如需转载请联系佰傲谷并在醒目位置注明出处﹀ 点亮在看，传递信息♥