People with HIV experience earlier impairments in physical function compared to people in the general population. They also exhibit an earlier presentation and more rapid development of frailty, a multisystemic syndrome of aging characterized by reduced activity, fatigue, slowness, weakness, and weight loss. While exercise can improve physical function in people with HIV, it is less effective in doing so than in the general population and is difficult to sustain in the long-term.
The goal of this clinical trial is to learn whether the medication tesamorelin will improve physical function and muscle health in adults with HIV when combined with exercise. Tesamorelin is a growth hormone-releasing hormone analogue that is FDA-approved to treat abdominal fat accumulation in people with HIV. While tesamorelin has also been shown to increase muscle mass and improve measures of muscle health, its effects on physical performance and muscle strength have not yet been evaluated.
During a 24-week intervention phase, half of participants will be randomly assigned to receive tesamorelin and half of participants will be randomly assigned to receive placebo (a look-alike substance that contains no drug). All participants also will engage in a home-based exercise intervention supervised by an exercise coach. During a subsequent 24-week extension phase, individuals will be monitored off study drug and supervised exercise, and be encouraged to continue to exercise independently.
The investigators will investigate effects of tesamorelin on physical function, muscle mass and quality, quality of life, and exercise adherence and self-efficacy. They also will evaluate whether effects of tesamorelin are maintained following treatment cessation. This study may identify an important strategy to improve how individuals aging with HIV function and feel with potential applications to other patient populations.

开始日期2025-03-01

申办/合作机构

The General Hospital Corp.

[+1]

NCT03375788

/ Active, not recruiting临床2期IIT

Growth Hormone Releasing Hormone Analog to Improve Nonalcoholic Fatty Liver Disease and Associated Cardiovascular Risk

Nonalcoholic fatty liver disease (NAFLD) is common in individuals with obesity and is a significant threat to public health, because it can lead to impaired liver function and liver failure. Growth hormone is a hormone produced in the pituitary gland that helps regulate metabolism and growth. Individuals with obesity, on average, secrete less growth hormone than individuals without obesity. There are data to suggest that growth hormone may help to reduce the amount of fat in the liver, and may also reduce inflammation in the liver, both of which would be helpful to individuals with NAFLD. The purpose of this study is to investigate whether treatment with a drug called tesamorelin, which is a growth hormone releasing hormone analogue, will decrease liver fat and improve liver inflammation and scarring in obese individuals with NAFLD.

开始日期2019-01-17

申办/合作机构

The General Hospital Corp.

NCT03226821

/ Recruiting临床4期IIT

Body Composition and Adipose Tissue in HIV Lipodystrophy: Effects of Tesamorelin Therapy

In this study, the investigators will examine the effect of therapy with the Growth Hormone Releasing Hormone (GHRH) analog tesamorelin on body composition in patients with HIV lipodystrophy and central adiposity. This study is a single arm prospective study of tesamorelin therapy of patients with HIV lipodystrophy. Subjects will do body composition testing, adipose tissue biopsy, metabolic rate measurements and insulin sensitivity assessment before, 6 and 12 months after daily injections of tesamorelin 2 mg by subcutaneous injection.

开始日期2018-02-07

申办/合作机构

Columbia University

[+1]

100 项与替莫瑞林相关的临床结果

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100 项与替莫瑞林相关的转化医学

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100 项与替莫瑞林相关的专利（医药）

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项与替莫瑞林相关的文献（医药）

2024-10-01·AIDS

Efficacy and safety of tesamorelin in people with HIV on integrase inhibitors

Article

作者: Toribio, Mabel ; Hadigan, Colleen M ; Fourman, Lindsay T. ; Johnson, Julia E. ; Arpante, Allison K. ; Ockene, Mollie W. ; Russo, Samuel C. ; Lee, Hang ; Erlandson, Kristine M ; Johnson, Julia E ; Hadigan, Colleen M. ; Grinspoon, Steven K. ; Stanley, Takara L. ; Arpante, Allison K ; Ockene, Mollie W ; Russo, Samuel C ; Grinspoon, Steven K ; Stanley, Takara L ; Fourman, Lindsay T

Objective:Tesamorelin is the only FDA-approved therapy to treat abdominal fat accumulation in people with HIV (PWH). Phase III clinical trials were conducted prior to the introduction of integrase inhibitors (INSTIs), which are now a mainstay of HIV antiretroviral therapy.Design:We leveraged a randomized double-blind trial of 61 PWH and metabolic dysfunction-associated steatotic liver disease to evaluate the efficacy and safety of tesamorelin 2 mg once daily vs. identical placebo among participants on INSTI-based regimens at baseline.Methods:In the parent clinical trial, visceral fat cross-sectional area, hepatic fat fraction, and trunk-to-appendicular fat ratio were quantified using magnetic resonance imaging, proton magnetic resonance spectroscopy, and dual-energy x-ray absorptiometry, respectively, at baseline and 12 months. Metabolic and safety outcomes were compared between treatment arms.Results:Among 38 participants on INSTI-based regimens at baseline, 15 individuals on tesamorelin and 16 individuals on placebo completed the 12-month study. Tesamorelin led to significant declines in visceral fat (median [interquartile range]: −25 [−93, −2] vs. 14 [3, 41] cm2, P = 0.001), hepatic fat (−4.2% [−12.3%, −2.7%] vs. −0.5% [−3.9%, 2.7%], P = 0.01), and trunk-to-appendicular fat ratio (−0.1 [−0.3, 0.0] vs. 0.0 [−0.1, 0.1], P = 0.03). Tesamorelin was well tolerated with a similar frequency of adverse events, including hyperglycemia, between groups.Conclusions:The current analysis provides the first dedicated data on the efficacy and safety of tesamorelin among PWH on INSTI-based regimens. Despite the association of INSTI use with weight gain and adipose tissue dysfunction, tesamorelin had beneficial effects on body composition with no exacerbation of glycemic control.

2024-08-12·Topics in antiviral medicine

CROI 2024: Neuropsychiatric Complications in People With HIV.

Review

作者: Letendre, Scott L ; Corley, Michael J ; Nightingale, Sam

The 2024 Conference on Retroviruses and Opportunistic Infections featured new and impactful findings about neuropsychiatric complications in people with HIV and other infections. Reports included new evidence from low- and middleincome countries, HIV persistence in the central nervous system, aging-related complications (including cerebrovascular disease), additional data relevant to pathogenesis, and therapeutics. Also included were new evidence of active HIV RNA transcription in cells from cerebrospinal fluid and the brain during virally suppressive antiretroviral therapy as well as links between neuropsychiatric complications or brain imaging findings in people with HIV and a) carotid artery inflammation and cerebrovascular disease, b) Alzheimer's disease genetic risk, c) social determinants of health, including exposure to pollution, and d) epigenetic aging. New therapeutic findings were presented on the cerebrospinal fluid inhibitory quotient, the effects of polypharmacy, and clinical trials of tesamorelin and telmisartan. This review summarizes these and other new findings and highlights new research directions for the neuro-HIV field.

2023-12-01·Analytical biochemistry

Cationic exchange SPE combined with triple quadrupole UHPLC-MS/MS for detection of GHRHs in urine samples.

Article

作者: David, Victor ; Cristea, Cătălina-Diana ; Radu, Mihai ; Toboc, Ani ; Stan, Cristina

The use of growth hormone-releasing hormones (GHRHs) is prohibited in sports according to the regulations of the World Anti-Doping Agency (WADA). Considering the complexity of urine samples and the low concentrations at which these analytes should be detected, analyzing GHRHs is a challenging task. In most of the studies, GHRHs are analyzed using UHPLC-HRMS with an orbitrap. The present developed and validated method for some GHRHs (tesamorelin, CJC-1295, sermorelin (GRF 1-29), sermorelin (3-29)-NH₂, somatorelin) is based on the triple quadrupole UHPLC/MS-MS method with solid phase extraction (SPE) with weak cation exchange and is able to detect concentrations as low as 0.2 ng/mL (LOD), a limit of quantification (LOQ) at 0.6 ng/mL, and linearity across the range of 0.1 ng/mL to 1.2 ng/mL. The present method developed by our doping control laboratory was validated according to WADA technical documents for selectivity, limit of detection (LOD), carryover, reliability of detection, stability and recovery. The results show that the method has adequate recoveries and sensitivity, hence, it can be employed for routine screening in anti-doping laboratories.

项与替莫瑞林相关的新闻（医药）

2025-01-10

·GlobeNewswire-Health Care

World Orphan Drug Alliance expands to Canada and full LATAM region, completing the first truly global commercialization offering

By adding Canada and the entire Latin America region, including Mexico, WODA completes its full global commercialization offering for rare and specialty therapies.With two new members - Innovative Medicines Group and Theratechnologies - WODA now provides access to over 210 million rare disease patients. MONTREAL and MONTEVIDEO, Uruguay, Jan. 10, 2025 (GLOBE NEWSWIRE) -- The World Orphan Drug Alliance (WODA), a global alliance of commercial distributors dedicated to providing access to treatments for rare diseases and specialty medicines in complex markets worldwide, has expanded its reach to include Canada and all of Latin America (LATAM). The addition of two new members - Theratechnologies Inc. for Canada and Innovative Medicines Group (IMG) for LATAM - brings the total number of countries covered by WODA to 158 and extends its reach to 210 million rare disease patients. The alliance now consists of 12 members, each being expert for the region covered. This expansion effectively completes WODA's ambition to provide global access to rare disease treatments. “With a new partner in LATAM and the addition of Canada, we are thrilled to become the only truly global-for-local platform," said Patrick Jordan, Chairman of WODA. "Canada not only gives our partners a footprint in North America, but with Theratechnologies’ operations in the U.S., they can be a U.S. solution as well. With IMG, we have a broader coverage of LATAM, including Mexico." Headquartered in Montreal, Theratechnologies was one of WODA’s first partners with the distribution of EGRIFTA SV® globally. They have recently broadened their portfolio, announcing an exclusive licensing deal with Ionis Pharmaceuticals for two assets in Canada. With a strong history of collaboration, Theratechnologies is a natural partner for WODA in Canada. WODA has also gained a strong new member in IMG, introducing a new platform for LATAM. IMG allows for a turnkey solution in the stalwart markets of Brazil, Argentina, and Colombia, and with a strong regional footprint ensures coverage across the rest of Latin America, including Mexico. Based in Montevideo, Uruguay, the team has a proven track record of serving rare disease patients throughout the region. The Alliance now provides a broad outreach with a local focus through thirteen separate entities: Specialised Therapeutics (Australia, New Zealand and Southeast Asia), Medis (Central and Eastern Europe & Nordics), Path Pharma (Greece, Cyprus, Malta), CTS (Israel), Innovative Medicines Group (Latin America), Vector Pharma (Middle East, North Africa, and Turkey), Orpharm (Russia and the CIS region), EffRx (Switzerland), Veld (Africa), Nxera (Japan, South Korea), Theratechnologies (Canada) and its boutique consultancy partner Decisive Consulting. About WODAThe World Orphan Drug Alliance (WODA) is a global alliance of commercial distributors focused on providing access to treatments for rare diseases and specialty medicines in complex markets around the world. WODA aims to provide comprehensive support to pharmaceutical and biotech companies with rare disease, oncology, and highly specialized therapeutics portfolio, starting from Named Patient Programs through to full commercialization. More about WODA: www.woda-alliance.com Media contact: Tina Vojnovic, tina.vojnovic@woda-alliance.com+386 41 744 735

引进/卖出孤儿药

2025-01-09

·GlobeNewswire-Health Care

Theratechnologies Provides Update on EGRIFTA SV® Supply

MONTREAL, Jan. 09, 2025 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that a shortage of EGRIFTA SV® (tesamorelin for injection) will occur at the patient level in mid-January 2025, following a voluntary shutdown of the contract manufacturing facility in 2024. While the Company is aware of remaining inventory of EGRIFTA SV® in certain pharmacies throughout the United States, several pharmacies have reported stockouts. Theratechnologies filed a required Prior Approval Supplement (PAS) to the U.S. Food and Drug Administration (FDA) on December 18, 2024. The Company subsequently submitted questions as part of a Type D meeting request concerning the status of two recently manufactured batches of EGRIFTA SV®, which are waiting to be released to pharmacies. The FDA confirmed that it would respond to the Company’s questions in writing no later than February 8, 2025. “We remain committed to people with HIV who rely on EGRIFTA SV®, as it is the only FDA-approved medicine of its kind, and we will continue to work with the FDA to expedite the release of the new batches for patients,” said Christian Marsolais, Ph.D., Senior Vice President and Chief Medical Officer at Theratechnologies. The Company will update the market on any further material developments. EGRIFTA SV® is distributed in the United States only. About Theratechnologies Theratechnologies (TSX: TH) (NASDAQ: THTX) is a biopharmaceutical company focused on the development and commercialization of innovative therapies addressing unmet medical needs. Further information about Theratechnologies is available on the Company's website at www.theratech.com, on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov. Follow Theratechnologies on Linkedin and Twitter. Forward-Looking Information This press release contains forward-looking statements and forward-looking information (collectively, the “Forward-Looking Statements”) within the meaning of applicable securities laws, that are based on management’s beliefs and assumptions and on information currently available to it. You can identify forward-looking statements by terms such as “may”, “will”, “should”, “could”, “promising”, “would”, “outlook”, “believe”, “plan”, “envisage”, “anticipate”, “expect” and “estimate”, or the negatives of these terms, or variations of them. The Forward-Looking Statements contained in this press release include, but are not limited to, statements regarding: (i) the time period related to the availability of EGRIFTA SV® to patients; (ii) the date by which the FDA will respond to the Company’s questions following its request to have a Type D meeting; and (iii) the timelines related to the potential release of the recently manufactured batches of EGRIFTA SV®. Although the Forward-Looking Statements contained in this press release are based upon what the Company believes are reasonable assumptions in light of the information currently available, investors are cautioned against placing undue reliance on these statements since actual results may vary from the Forward-Looking Statements contained in this press release. Certain assumptions made in preparing the Forward-Looking Statements include that: (i) the FDA will provide answers to the questions asked by the Company by February 8, 2025; (ii) the responses to the questions asked by the Company will confirm that the Company is able to release the recently manufactured batches of EGRIFTA SV® or will confirm that the review of the PAS will be completed prior to the expiry of a four-month period; (iii) current market demand for EGRIFTA SV® will remain unaffected; (iv) the shortage of EGRIFTA SV® will not adversely impact the financial conditions of the Company; and (v) the Company will be able to negotiate waivers of defaults with its secured creditors to the extent the drug shortage period results in defaults under its credit agreements. Forward-Looking Statements assumptions are subject to a number of risks and uncertainties, many of which are beyond the Company’s control, that could cause actual results to differ materially from those that are disclosed in or implied by such Forward-Looking Statements. These risks and uncertainties include, but are not limited to: (i) delays in responding to the Company’s questions beyond February 8, 2025; (ii) refusal by the FDA to release the recently manufactured batches of EGRIFTA SV® until the PAS review is completed; (iii) FDA’s review of the PAS not being completed before April 18, 2025; (iv) non-approval of the PAS; (v) issuance of questions as part of the PAS review by the FDA resulting in delays in completing the PAS review and resulting in delays beyond April 18, 2025 to release the recently manufactured batches of EGRIFTA SV®; (vi) issuance of a complete response letter following the filing of the PAS as a result of the manufacturing site being classified as Official Action Indicated preventing the Company from releasing the recently manufactured batches of EGRIFTA SV®; (vii) a decrease in demand for EGRIFTA SV® due to its shortage potentially adversely impacting the resumption of the commercialization of EGRIFTA SV®; (viii) losses of key employees due to the shortage; and (ix) defaults under the covenants of the credit agreements resulting from the shortage. The Company refers current and potential investors to the “Risk Factors” section of the Company’s Form 20-F dated February 21, 2024 available on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov under Theratechnologies’ public filings. The reader is cautioned to consider these and other risks and uncertainties carefully and not to put undue reliance on forward-looking statements. Forward-Looking Statements reflect current expectations regarding future events and speak only as of the date of this press release and represent the Company’s expectations as of that date. The Company undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise, except as may be required by applicable law. Contacts: Investor inquiries:Joanne ChoiSenior Director, Investor Relationsjchoi@theratech.com1-551-261-0401 Media inquiries:Julie SchneidermanSenior Director, Communications & Corporate Affairscommunications@theratech.com1-514-336-7800

上市批准加速审批

2025-01-06

·CPHI制药在线

FDA 2025年第一季度审批全览

关注并星标CPHI制药在线 01 Ozempic (semaglutide) ● 开发商：诺和诺德 ● 模态：多肽 ● PDUFA日期：2025年1月 ● 适应症类型/说明：代谢、肾脏/每周一次皮下注射semaglutide的新适应症，用于治疗患有 2 型糖尿病和慢性肾脏病 (CKD) 的成年人，包括延缓 CKD 进展并降低肾脏和心血管死亡风险。 ● 审评类型：标准审评 ● 申请递交时间：2024年3月 ● 审评情况：FDA于2024年6月24日接受申请 02 Subvenite (lamotrigine oral liquid) ● 开发商：OWP Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年1月3日 ● 适应症类型/说明：中枢神经系统疾病/用于治疗癫痫和躁郁症的抗癫痫药物的新型口服液制剂 ● 审评类型：505(b)(2) NDA标准审评 ● 申请递交时间：2024年3月 ● 审评情况：FDA于2024年5月20日接受申请 03 TX05 (trastuzumab 生物类似药) ● 开发商：Tanvex ● 模态：单抗 ● PDUFA日期：不晚于2025年1月6日 ● 适应症类型/说明：癌症/Genetech的 HER2 靶向抗体Herceptin的生物类似药 ● 审评类型：351(k) BLA标准审评 ● 申请递交时间：2024年7月6日（重新递交） ● 审评情况：FDA之前于2022年8月签发了完整回复函 04 Ryoncil (remestemcel-L-rknd，新生物制品) ● 开发商：Mesoblast ● 模态：细胞疗法 ● PDUFA日期：2025年1月7日 ● 适应症类型/说明：免疫学/同种异体扩增间充质基质细胞 (MSC) 干细胞疗法，用于治疗类固醇难治性急性移植物抗宿主病 (SR-aGVHD) 的儿科患者 ● 审评类型：优先审评 ● 申请递交时间：2024年7月8日（重新递交） ● 审评情况：FDA之前于2022年8月3日签发了第二封完整回复函（第一封签发于2020年10月1日） 05 Tabelecleucel （新生物制品） ● 开发商：Atara Biotherapeutics/Pierre Fabre ● 模态：细胞疗法 ● PDUFA日期：2025年1月15日 ● 适应症类型/说明：免疫学、支持性治疗/同种异体、Epstein-Barr 病毒 (EBV) 特异性 T 细胞免疫疗法，用于单药治疗 2 岁及以上患有 EBV 阳性移植后淋巴增生性疾病 (EBV+ PTLD) 的成人和儿童患者，这些患者至少接受过一次先前治疗 ● 审评类型：优先审评 ● 申请递交时间：2024年5月20日 ● 审评情况：突破性疗法认定 06 Lumakras (sotorasib) ● 开发商：安进 ● 模态：小分子 ● PDUFA日期：2025年1月17日 ● 适应症类型/说明：癌症 /新适应症，与panitumumab (Vectibix) 组成联合疗法，用于治疗 KRAS G12C 突变转移性结直肠癌 (CRC) 。 ● 审评类型：优先审评 ● 申请递交时间：2024年4月 ● 审评情况：延期，原PDUFA日期为2024年10月17日 07 Leqembi (lecanemab-irmb) ● 开发商：卫材/渤健 ● 模态：抗体 ● PDUFA日期：2025年1月25日 ● 适应症类型/说明：中枢神经系统疾病/新的维持给药方案，为已完成每两周一次的阿尔茨海默病治疗初始阶段的患者提供每月一次的Leqembi静脉注射 ● 审评类型：标准审评 ● 申请递交时间：2024年4月1日 ● 审评情况：FDA于2024年6月9日接受申请 08 CT-P47 (tocilizumab生物类似药) ● 开发商：Celltrion ● 模态：单抗 ● PDUFA日期：不晚于2025年1月28日 ● 适应症类型/说明：免疫学/抗白细胞介素 6 (IL-6) 抗体Actemra生物类似药，针对类风湿关节炎和幼年特发性关节炎等适应症 ● 审评类型：351(k) BLA标准审评 ● 申请递交时间：2024年1月28日 09 Elamipretide （新分子实体） ● 开发商：Stealth BioTherapeutics ● 模态：多肽 ● PDUFA日期：2025年1月29日 ● 适应症类型/说明：心血管先天性疾病/与磷脂心磷脂结合的线粒体保护肽，用于治疗巴特综合征 ● 审评类型：优先审评（最初为标准审评，但在Stealth Bio的要求下更改为优先审评） ● 申请递交时间：2024年1月 ● 审评情况：最初申请递交时间为2021年8月，FDA 2021年10月签署拒绝备案(RTF)信函；重新递交后，FDA于2024年4月8日接受申请；罕见儿科疾病认定。 10 Suzetrigine (VX-548，新分子实体) ● 开发商：Vertex ● 模态：小分子 ● PDUFA日期：2025年1月30日 ● 适应症类型/说明：中枢神经系统疾病/选择性 NaV1.8 疼痛信号抑制剂，口服、非阿片类药物，治疗中度至重度急性疼痛 ● 审评类型：优先审评 ● 申请递交时间：2024年5月30日 ● 审评情况：FDA 2024年7月30日接受申请；突破性疗法认定 11 Treosulfan (新药) ● 开发商：medac GmbH/Medexus ● 模态：小分子 ● PDUFA日期：2025年1月30日 ● 适应症类型/说明：癌症、血液疾病/双功能烷化剂，与氟达拉滨联合使用，作为接受异基因造血干细胞移植 (allo-HSCT) 的成人和儿科患者的减毒预处理方案 ● 审评类型：标准审评 ● 申请递交时间：2024年4月 ● 审评情况：FDA 2021年8月3日签署完整回复函；重新提交申请后FDA于2024年6月6日接受申请；PDUFA日期由最初的2024年10月30日推迟。 12 AXS-07 (meloxicam/rizatriptan) ● 开发商：Axsome Therapeutics ● 模态：小分子 ● PDUFA日期：2025年1月31日 ● 适应症类型/说明：中枢神经系统疾病/偏头痛的急性治疗 ● 审评类型：505(b)(2) NDA标准审评 ● 申请递交时间：2024年7月 ● 审评情况：2021 年 7 月首次提交申请，FDA 于 2022 年 5 月 2 日签署完整回复函；2024 年 9 月 4 日FDA接受重新提交审查。 13 Translarna (ataluren，新分子实体) ● 开发商：PTC Therapeutics ● 模态：小分子 ● PDUFA日期：2025年2月或3月 ● 适应症类型/说明：中枢神经系统疾病/治疗无义突变杜氏肌营养不良症 (nmDMD) ● 审评类型：优先审评 ● 申请递交时间：2024年8至9月 14 Omvoh (mirikizumab-mrkz) ● 开发商：礼来 ● 模态：单抗 ● PDUFA日期：不迟于2025年2月 ● 适应症类型/说明：胃肠道疾病/ Omvoh的新适应症，治疗患有中度至重度活动性克罗恩病的成年人 ● 审评类型：标准审评 ● 申请递交时间：2024年3至4月 15 Enhertu (fam-trastuzumab deruxtecan-nxki) ● 开发商：第一三共/阿斯利康 ● 模态：ADC ● PDUFA日期：2025年2月1日 ● 适应症类型/说明：癌症/Enhertu新适应症，治疗不可切除或转移性 HER2 低或 HER2 超低乳腺癌的成年人 ● 审评类型：优先审评 ● 申请递交时间：2024年8月1日 ● 审评情况： FDA 于 2024年10月1日接受申请；突破性疗法认定 16 SPN-830 (apomorphine infusion pump) ● 开发商：Supernus/US WorldMeds/Brittania ● 模态：小分子 ● PDUFA日期：2025年2月1日 ● 适应症类型/说明：中枢神经系统疾病/多巴胺激动剂apomorphine的持续皮下输注制剂，用于治疗帕金森病成人的运动波动（OFF 发作） ● 审评类型：标准审评 ● 申请递交时间：2024年8月1日 ● 审评情况：经历一次RTF（Refuse to File）和两次完整回复函。 17 Bentracimab （新生物制品） ● 开发商：SFJ Pharmaceuticals/SERB Pharmaceuticals ● 模态：抗体 ● PDUFA日期：不晚于2025年2月2日 ● 适应症类型/说明：心血管疾病/针对因使用替格瑞洛而引起的紧急出血事件，或在紧急手术情况下需要逆转替格瑞洛的抗血小板效果 ● 审评类型：优先审评 ● 申请递交时间：2024年5至6月 ● 审评情况：FDA 2024年8月2日接受BLA申请；突破性疗法认定 18 Datopotamab deruxtecan (Dato-DXd，新生物制品) ● 开发商：第一三共/阿斯利康 ● 模态：ADC ● PDUFA日期：不晚于2025年2月2日 ● 适应症类型/说明：癌症/转移性激素受体 (HR) 阳性、HER2 阴性（IHC 0、IHC 1+ 或 IHC 2+/ISH-）乳腺癌成人患者 ● 审评类型：标准审评 ● 申请递交时间：2024年1至2月 ● 审评情况：FDA 2024年4月2日接受BLA申请 19 Sublocade (buprenorphine depot injection) ● 开发商：Indivior ● 模态：小分子 ● PDUFA日期：2025年2月7日 ● 适应症类型/说明：中枢神经系统疾病/皮下注射阿片类激动剂，用于治疗中度至重度阿片类药物使用障碍 (OUD) ● 审评类型：优先审评 ● 申请递交时间：2024年8月 ● 审评情况：FDA 2024年10月7日接受申请 20 Rexulti (brexpiprazole) ● 开发商：Otsuka/Lundbeck ● 模态：小分子 ● PDUFA日期：2025年2月8日 ● 适应症类型/说明：中枢神经系统疾病/血清素-多巴胺活性调节剂 (SDAM) 非典型抗精神病药，新适应症，与舍曲林联合使用，用于治疗成人创伤后应激障碍 (PTSD) ● 审评类型：标准审评 ● 申请递交时间：2024年4月10日 ● 审评情况：FDA 2024年6月25日接受申请 21 CHIKV VLP (chikungunya vaccine, 此前称作PXVX0317，新生物制品) ● 开发商：Bavarian Nordic ● 模态：疫苗 ● PDUFA日期：2025年2月14日 ● 适应症类型/说明：传染病/用于成人和 12 岁及以上的儿童，预防基孔肯雅病毒感染 ● 审评类型：优先审评 ● 申请递交时间：2024年6月17日 ● 审评情况：突破性疗法认定 22 MenABCWY (meningococcal group A,B,C,Y/W-135 vaccine，新生物制品) ● 开发商：GSK ● 模态：疫苗 ● PDUFA日期：2025年2月14日 ● 适应症类型/说明：传染病/预防侵袭性脑膜炎球菌病 ● 审评类型：标准审评 ● 申请递交时间：2024年2月 ● 审评情况：FDA 2024年4月16日接受申请 23 Vimseltinib （新分子实体） ● 开发商：Deciphera (Ono Pharmaceutical) ● 模态：小分子 ● PDUFA日期：2025年2月17日 ● 适应症类型/说明：癌症/治疗腱鞘巨细胞瘤 (TGCT) 患者 ● 审评类型：优先审评 ● 申请递交时间：2024年6月 ● 审评情况：FDA 2024年8月15日接受申请 24 Mirdametinib （新分子实体） ● 开发商：SpringWorks Therapeutics ● 模态：小分子 ● PDUFA日期：2025年2月18日 ● 适应症类型/说明：癌症/治疗患有 1 型神经纤维瘤病相关丛状神经纤维瘤 (NF1-PN) 的儿童和成人患者 ● 审评类型：优先审评 ● 申请递交时间：2024年6月 ● 审评情况：FDA 2024年8月15日接受申请 25 ET-400 (hydrocortisone oral solution) ● 开发商：Eton Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年2月28日 ● 适应症类型/说明：内分泌、免疫学/氢化皮质酮(又称氢化可的松)，新型糖皮质激素口服溶液剂型，在室温下稳定，适用于婴儿和儿科患者 ● 审评类型：505(b)(2) NDA标准审评 ● 申请递交时间：2024年4月30日 ● 审评情况：FDA 2024年7月15日接受申请 26 Adcetris (brentuximab vedotin) ● 开发商：辉瑞 ● 模态：ADC ● PDUFA日期：2025年3月 ● 适应症类型/说明：癌症/CD30 靶向 ADC brentuximab vedotin 的新适应症，用于与来那度胺和利妥昔单抗联合治疗复发或难治性大 B 细胞淋巴瘤 (LBCL) ● 审评类型：标准审评 ● 申请递交时间：2024年5月 ● 审评情况：FDA 2024年7月30日接受申请 27 Susvimo (眼部植入物玻璃体注射 ranibizumab) ● 开发商：罗氏 ● 模态：单抗 ● PDUFA日期：不晚于2025年3月 ● 适应症类型/说明：眼科/ranibizumab的新适应症，该植入物可提供定制配方的 VEGF 抑制剂（不同于 Lucentis 玻璃体内注射），最长可使用六个月，用于治疗糖尿病性黄斑水肿和糖尿病性视网膜病变 ● 审评类型：标准审评 ● 申请递交时间：2024年5月 ● 审评情况：FDA 2024年7月18日接受申请 28 neffy (肾上腺素 1mg 鼻喷雾剂) ● 开发商：ARS Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年3月6日 ● 适应症类型/说明：免疫学/1mg 新浓度和扩大的儿科适应症，适用于体重 15-30 公斤儿童的 I 型过敏反应（包括过敏性休克）的鼻内紧急治疗 ● 审评类型：优先审评；505(b)(2) NDA ● 申请递交时间：2024年9月9日 29 Furoscix (furosemide, 皮下制剂) ● 开发商：scPharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年3月6日 ● 适应症类型/说明：心血管疾病/新适应症，皮下注射，治疗慢性肾病 (CKD) 患者因液体超负荷引起的水肿 ● 审评类型：标准审评 ● 申请递交时间：2024年5月 ● 审评情况：FDA 2024年7月25日接受申请 30 CT-P39 (omalizumab生物类似药) ● 开发商：Celltrion ● 模态：单抗 ● PDUFA日期：不晚于2025年3月11日 ● 适应症类型/说明：免疫学/Genetech的Xolair 的可互换生物类似药，用于治疗严重持续性过敏性哮喘、鼻息肉、荨麻疹和免疫球蛋白 E 介导的食物过敏等。 ● 审评类型：标准审评；351(k) BLA ● 申请递交时间：2024年3月11日 31 Condoliase (SI-6603，新生物制品) ● 开发商：Seikagaku/Ferring Pharmaceutical ● 模态：蛋白质 ● PDUFA日期：不晚于2025年3月14日 ● 适应症类型/说明：中枢神经系统疾病/成人腰椎间盘突出症 (LDH) 相关的根性腿痛 ● 审评类型：标准审评 ● 申请递交时间：2024年3月 ● 审评情况：FDA 2024年5月14日接受申请，2025年1月1日FDA顾问委员会会议 32 Revakinagene taroretcel (NT-501，新生物制品) ● 开发商：Neurotech Pharmaceuticals ● 模态：基因疗法 ● PDUFA日期：2025年3月18日 ● 适应症类型/说明：眼科/治疗 2 型黄斑毛细血管扩张症（MacTel，即特发性近焦点黄斑毛细血管扩张症） ● 审评类型：优先审评 ● 申请递交时间：2024年4月 ● 审评情况：FDA 2024年6月20日接受申请，PDUFA日期从最初的2024年12月17日延期 33 Camrelizumab (SHR-1210，新生物制品) ● 开发商：江苏恒瑞 (2023年10月授权Elevar) ● 模态：单抗 ● PDUFA日期：2025年3月20日 ● 适应症类型/说明：癌症/PD-1 抑制剂，与 Elevar 的 VEGFR-2 激酶抑制剂rivoceranib联合用于不可切除或转移性肝细胞癌 (uHCC) 的一线全身治疗 ● 审评类型：标准审评 ● 申请递交时间：2024年9月23日 ● 审评情况：首次BLA申请2023年5月17日，FDA 2024年5月27日签发完全回复函；再次申请获得接受。 34 Amvuttra (vutrisiran) ● 开发商：Alnylam ● 模态：寡核苷酸 ● PDUFA日期：2025年3月23日 ● 适应症类型/说明：心血管疾病、中枢神经系统疾病/ vutrisiran 新适应症，治疗伴有心肌病的转甲状腺素蛋白淀粉样变性 (ATTR-CM) ● 审评类型：使用一张优先审评券PRV ● 申请递交时间：2024年10月9日 ● 审评情况：FDA 2024年11月25日接受申请。 35 Rivoceranib （新分子实体） ● 开发商：Elevar Therapeutics (HLB LifeScience) ● 模态：小分子 ● PDUFA日期：不晚于2025年3月23日 ● 适应症类型/说明：癌症/与恒瑞医药的 PD-1 抑制剂Camrelizumab联合用于不可切除的肝细胞癌 (uHCC) 的一线治疗 ● 审评类型：标准审评 ● 申请递交时间：2024年9月23日 ● 审评情况：2023年5月17日递交的NDA申请被拒，再申请获得接受。 36 TLX007-CDx ● 开发商：Telix ● 模态：放射性示踪剂 ● PDUFA日期：2025年3月24日 ● 适应症类型/说明：癌症成像剂/前列腺癌诊断 ● 审评类型：标准审评 ● 申请递交时间：2024年5月27日 ● 审评情况：FDA 2024年7月24日接受申请。 37 Gepotidacin （新分子实体） ● 开发商：GSK ● 模态：小分子 ● PDUFA日期：2025年3月26日 ● 适应症类型/说明：传染病/口服治疗患有无并发症尿路感染 (uUTI) 的成年女性（≥40 公斤）和青少年（≥12 岁，≥40 公斤） ● 审评类型：优先审评 ● 申请递交时间：2024年7月 ● 审评情况：FDA 2024年10月16日接受申请。 38 Cardamyst (etripamil，新分子实体) ● 开发商：Milestone Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年3月26日 ● 适应症类型/说明：心血管疾病/鼻腔喷雾剂，治疗阵发性室上性心动过速 (PSVT) ● 审评类型：标准审评 ● 申请递交时间：2024年3月28日 ● 审评情况：FDA 2023年12月签发RTF信函，之后重新递交申请，FDA 2024年5月26日接受申请。 39 Egrifta MDV (tesamorelin F8 formulation) ● 开发商：Theratechnologies ● 模态：多肽 ● PDUFA日期：不晚于2025年3月26日 ● 适应症类型/说明：代谢疾病/用于治疗 HIV 相关脂肪营养障碍 ● 审评类型：标准审评 ● 申请递交时间：2024年11月26日 ● 审评情况：FDA接受之前遭受一份完整回复函。 40 DCCR (diazoxide choline) ● 开发商：Soleno Therapeutics ● 模态：小分子 ● PDUFA日期：2025年3月27日 ● 适应症类型/说明：内分泌疾病/治疗 4 岁及以上患有贪食症的普拉德-威利综合征 (PWS) 患者 ● 审评类型：优先审评 ● 申请递交时间：2024年6月27日 ● 审评情况：最初PDUFA日期为2024年12月27日，延期。 41 Fitusiran （新分子实体） ● 开发商：赛诺菲 ● 模态：寡核苷酸 ● PDUFA日期：2025年3月28日 ● 适应症类型/说明：血液疾病/成人和青少年A型或B型血友病 A 或 B 患者 ● 审评类型：标准审评 ● 申请递交时间：2024年3月 ● 审评情况：B型血友病疗法获得突破性疗法认定。 42 Darzalex Faspro (daratumumab/ hyaluronidase-fihj) ● 开发商：强生/Genmab ● 模态：抗体/酶 ● PDUFA日期：不晚于2025年3月30日 ● 适应症类型/说明：癌症/使用 Halozyme 的 Enhanze 技术皮下注射daratumumab，用于治疗新诊断的多发性骨髓瘤 (NDMM) 成人患者。 ● 审评类型：优先审评 ● 申请递交时间：2024年9月30日 43 Sepiapterin (PTC923, 最初称为CNSA-001，新分子实体) ● 开发商：PTC Therapeutics ● 模态：小分子 ● PDUFA日期：不晚于2025年3月30日 ● 适应症类型/说明：先天性缺陷/治疗儿童和成人苯丙酮尿症 (PKU) 患者，包括所有年龄段和疾病亚型 ● 审评类型：标准审评 ● 申请递交时间：2024年7月30日 ● 审评情况：FDA 2024年10月1日接受申请。 44 Jynneos (smallpox/monkeypox 疫苗) ● 开发商：Bavarian Nordic ● 模态：疫苗 ● PDUFA日期：不晚于2025年3月31日 ● 适应症类型/说明：传染病/基于减毒活痘苗病毒的新型冻干非复制型疫苗配方，适合长期储存，用于预防 18 岁及以上成年人的天花和猴痘 ● 审评类型：标准审评 ● 申请递交时间：2024年5月31日 Ref. Pink Sheet User Fee Goals Date. Retrieved on 02. 01. 2025. END 【智药研习社近期直播】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

优先审批免疫疗法生物类似药细胞疗法突破性疗法

100 项与替莫瑞林相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	替莫瑞林	H01AC06	DB08869

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
HIV感染	美国	Theratechnologies, Inc.	2010-11-10
脂肪营养不良	美国	Theratechnologies, Inc.	2010-11-10

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
肝纤维化	临床2期	加拿大	Theratechnologies Europe Ltd.	2022-01-30
肌肉萎缩	临床2期	-	Theratechnologies, Inc.	2011-09-01
慢性阻塞性肺疾病	临床2期	-	Theratechnologies, Inc.	2011-09-01
HIV血清阳性	药物发现	加拿大	Theratechnologies, Inc.	2008-05-01
HIV相关性脂肪营养不良综合征	药物发现	加拿大	Theratechnologies, Inc.	2005-06-01
HIV相关性脂肪营养不良综合征	药物发现	美国	Theratechnologies, Inc.	2005-06-01
2型糖尿病	药物发现	-	Theratechnologies, Inc.	2002-02-01

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


GlobeNewswire 人工标引	临床3期	心血管疾病 \| HIV感染	-	Tesamorelin	蓋願鬱壓艱壓襯範觸觸(繭顧膚觸鬱築築憲鬱構) = 鬱鏇製繭觸膚鏇廠獵選顧憲遞衊願網窪鹽襯願 (壓簾衊範選廠蓋壓網鬱, -0.89 ~ 0.05)	积极	2024-10-17
ENDO2021	N/A	代谢功能障碍相关的脂肪肝病维持 C-C Motif Chemokine Ligands 3 \| interleukin-8 \| interleukin-10 ... 更多	61	Tesamorelin	襯醖膚顧糧鑰繭餘鹽繭(網積鬱簾構顧膚蓋憲鬱) = 製鬱淵膚艱淵選廠衊衊遞蓋觸艱鹽糧衊製餘糧 (積醖網願憲艱窪網鏇鑰 ) 更多	积极	2021-05-03
NCT02196831 (CTgov)	N/A	非酒精性脂肪性肝炎 \| HIV感染	61	tesamorelin (Tesamorelin)	遞餘觸廠齋襯艱鏇積醖(築選窪艱獵襯繭壓淵積) = 壓遞鹹顧壓憲製積鏇繭衊鹹觸鹽鑰鑰齋範窪顧 (獵積壓鏇簾觸鹽糧衊製, 顧齋齋艱鹹觸鬱襯簾憲 ~ 糧襯憲鹽窪糧膚憲鹹獵) 更多	-	2020-01-13
				Placebo (Placebo)	遞餘觸廠齋襯艱鏇積醖(築選窪艱獵襯繭壓淵積) = 顧鹹繭鬱選鹹鏇鹽膚網衊鹹觸鹽鑰鑰齋範窪顧 (獵積壓鏇簾觸鹽糧衊製, 顧齋遞膚觸糧網遞憲衊 ~ 繭鹹構壓齋艱夢顧獵願) 更多
NCT00608023 \| NCT00123253 (Pubmed)	临床3期	HIV感染	806	Tesamorelin	(蓋鹹繭醖餘憲蓋鑰積鬱) = 壓窪顧願鹽餘鹽構獵鏇鹹範繭遞築醖廠淵築廠 (鬱網糧醖鬱鏇窪膚選鹽 ) 更多	-	2017-10-23
				Placebo	(蓋鹹繭醖餘憲蓋鑰積鬱) = 衊憲簾壓製製糧繭鹹窪鹹範繭遞築醖廠淵築廠 (鬱網糧醖鬱鏇窪膚選鹽 ) 更多
NCT00807365 (PP2, CTgov)	临床2期	-	5	GHRH	艱顧襯膚廠鏇夢遞夢鹹(範蓋鑰齋範淵鬱衊醖構) = 構憲鬱窪夢遞願餘願鹽簾餘構鹹餘選壓選築鬱 (繭積範築鬱製壓鹽艱遞, 衊齋鏇遞構壓鹽衊淵築 ~ 艱夢構簾衊範願壓觸繭) 更多	-	2017-04-27
NCT01263717 (CTgov)	N/A	HIV相关性脂肪营养不良综合征	54	tesamorelin (Tesamorelin)	製餘醖積繭蓋鬱構齋襯(願鏇選醖積窪壓淵範艱) = 鏇夢窪壓鬱顧艱觸顧遞襯衊築鬱鬱鹽網艱鏇鹽 (壓觸衊鏇憲廠齋鬱餘壓, 顧構鑰築築鑰壓糧鹽願 ~ 廠簾淵餘繭築艱齋觸窪) 更多	-	2014-10-13
				placebo (Placebo (Inactive Injection))	製餘醖積繭蓋鬱構齋襯(願鏇選醖積窪壓淵範艱) = 簾鏇構鑰顧網淵窪艱獵襯衊築鬱鬱鹽網艱鏇鹽 (壓觸衊鏇憲廠齋鬱餘壓, 顧襯鏇醖窪艱鏇構衊醖 ~ 夢願顧獵醖鹽襯網繭鑰) 更多
NCT01263717 (Pubmed \| JAMA)	N/A	HIV感染	50	Tesamorelin 2 mg	構鏇壓鏇願艱壓壓餘繭(繭衊蓋蓋遞網醖夢顧選) = 選鏇範蓋壓獵獵繭膚選餘膚鏇繭鹹夢構範範網 (醖艱築醖築鏇艱衊壓積 ) 更多	-	2014-07-30
				Placebo	構鏇壓鏇願艱壓壓餘繭(繭衊蓋蓋遞網醖夢顧選) = 糧夢淵襯襯鹹淵壓願構餘膚鏇繭鹹夢構範範網 (醖艱築醖築鏇艱衊壓積 ) 更多
NCT01388920 (CTgov)	临床2期	慢性阻塞性肺疾病 \| 肌肉萎缩	3	Tesamorelin (Tesamorelin 2 mg)	蓋選鏇夢築範艱顧網窪(艱鹹壓衊觸窪糧簾糧衊) = 齋壓醖壓膚夢糧網願鬱鹹鏇觸鬱網膚願鹹顧衊 (範衊鏇窪糧艱艱膚蓋蓋, 鬱鏇構鹽糧襯鹹構繭觸 ~ 壓醖膚築壓夢構夢獵構) 更多	-	2014-05-07
				Tesamorelin (Tesamorelin 3 mg)	蓋選鏇夢築範艱顧網窪(艱鹹壓衊觸窪糧簾糧衊) = 簾壓積顧蓋襯醖鏇範廠鹹鏇觸鬱網膚願鹹顧衊 (範衊鏇窪糧艱艱膚蓋蓋, 簾鹽簾鏇蓋鹽壓選壓顧 ~ 鹽鹹鬱鹹糧願顧範範繭) 更多
NCT00795210 (CTgov)	N/A	脂肪营养不良 \| HIV感染	25	growth hormone (GH 6mcg/kg/d)	壓醖觸網夢壓鏇醖繭衊(鏇齋簾夢鬱觸壓夢鏇範) = 醖遞糧觸觸範築餘壓鏇鬱鑰廠鏇範膚艱繭積觸 (鑰鹹夢鹹積淵網願製齋, 膚築憲繭鑰鹽餘獵繭艱 ~ 選齋鏇廠製廠簾淵積糧) 更多	-	2014-01-08
				growth hormone (GH 2mg Daily)	壓醖觸網夢壓鏇醖繭衊(鏇齋簾夢鬱觸壓夢鏇範) = 範壓齋醖醖鏇積鑰鬱餘鬱鑰廠鏇範膚艱繭積觸 (鑰鹹夢鹹積淵網願製齋, 遞鬱選糧窪製鏇鬱鑰獵 ~ 壓膚簾膚膚選鏇蓋衊膚) 更多