Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few published reports on the effects of treatment on physical function in patients with SCD. The hypothesis to be tested is that anemic SCD patients will have improvements in performance after 6 months of voxelotor treatment.

开始日期2023-10-23

申办/合作机构

Inova Health Care Services

[+1]

NCT05228821

/ Withdrawn临床4期

An Open Label, Single Arm, Multicenter Study to Evaluate the Effect of Voxelotor on Cerebral Blood Flow and Neurocognitive Function in Adolescents and Adults With Sickle Cell Disease

This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD).

开始日期2023-08-09

申办/合作机构

Pfizer Inc.

NCT05981365

/ Completed临床1期

A Phase 1, Open-Label, Two-Part, Fixed-Sequence, Drug-Drug Interaction Study to Evaluate the Effect of Voxelotor on the Pharmacokinetics of Selected CYP and Transporter Probe Substrates in Healthy Participants

This research study is examining multiple doses of voxelotor (a study drug intended for treatment of sickle cell disease) and how it interacts with additional substrates (substrates are drugs or other substances that are metabolized by cytochrome enzymes. The substrates used in this study are FDA approved medications). The study will help to determine the safety and tolerability of the study drugs taken together, as well as the pharmacokinetics (PK) on how your body processes and responds to the combination of the study drug and substrates. Although these drugs are FDA approved, their use in this study is experimental.

开始日期2023-04-17

申办/合作机构

Pfizer Inc.

100 项与伏塞洛托相关的临床结果

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100 项与伏塞洛托相关的转化医学

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100 项与伏塞洛托相关的专利（医药）

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191

项与伏塞洛托相关的文献（医药）

2025-05-01·LIFE SCIENCES

A critique review of fetal hemoglobin modulators through targeting epigenetic regulators for the treatment of sickle cell disease

Review

作者: Ala, Chandu ; Ramalingam, Sivaprakash ; Sankaranarayanan, Murugesan ; Kondapalli Venkata Gowri, Chandra Sekhar

Sickle cell disease (SCD) is one of the most prevalent hereditary blood disorders characterized by aberrant hemoglobin synthesis that causes red blood cells (RBCs) to sickle and result in vaso-occlusion. The complex pathophysiological mechanisms that underlie SCD are explored in this study, including hemoglobin polymerization, the formation of fetal hemoglobin (HbF), and hemoglobin switching regulation. Notably, pharmaceutical approaches like hydroxyurea, l-glutamine, voxelotor, and crizanlizumab, in addition to therapeutic techniques like gene therapies like Casgevy and Lyfgenia, signify noteworthy advancements in the management of issues connected to SCD. Furthermore, the deciphering of the molecular mechanisms that dictate hemoglobin switching has revealed several potentially therapeutic targets, including key transcriptional repressors such as β-cell lymphoma/leukemia 11A (BCL11A), Zinc finger and BTB domain-containing 7A (ZBTB7A), Nuclear Factor IX (NFIX), and Nuclear Factor IA (NFIA), which play crucial roles in γ-globin silencing. Additionally, transcriptional activators such as Nuclear Factor Y (NF-Y), and Hypoxia-inducible factor 1α (HIF1α) have emerged as promising regulators that can disrupt repression and enhance HbF synthesis. Other epigenetic regulators, such as lysine-specific histone demethylase 1 (LSD1), euchromatic histone methyltransferases 1/2 (EHMT1/2), histone deacetylases (HDACs), DNA methyltransferases (DNMTs), and protein arginine methyltransferases (PRMTs). It has been demonstrated that inhibiting these targets can prevent the silencing of the gene encoding for the formation of γ-chains and, in turn, increase the synthesis of HbF, providing a possible treatment option for SCD symptoms. These approaches could pave the way for innovative, mechanism-driven therapies that address the unmet medical needs of SCD patients.

2025-02-15·Clinical and experimental pediatrics

Promising role of voxelotor in managing sickle cell disease in children: a narrative review

Article

作者: Singh, Japna ; Agrawal, Amit ; Jadon, Gaurav ; Janjua, Dalwinder

Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent vasoocclusive crises. To date, 4 disease-modifying drugs have been approved for the treatment of SCD: hydroxyurea (an S-phase inhibitor), L-glutamine (an amino acid), crizanlizumab (a P-selectin inhibitor), and voxelotor (a hemoglobin S polymerization inhibitor). Preclinical studies suggested that voxelotor effectively treats SCD and sickle cell anemia (SCA). In a phase III trial, voxelotor-treated patients showed significantly elevated hemoglobin levels (>1 g/dL from baseline) compared to placebo-treated patients. The group that received voxelotor also showed a greater decrease in hemolytic markers but a comparable incidence of side effects. Six ongoing clinical trials also sought to ascertain the effectiveness and safety of high-dose voxelotor when administered to children younger than 12 years. Studies assessing their long-term efficacy and safety are needed to fully understand the role of voxelotor in treating SCD/SCA. In this review, we discuss the mechanisms, trials to date, and future treatment directions of voxelotor.

2025-01-01·AMERICAN JOURNAL OF HEMATOLOGY

Effect of voxelotor on cerebral perfusion and cerebral oxygen metabolism and cardiac stress in adult patients with sickle cell disease

Article

作者: Biemond, Bart J. ; Nederveen, Aart J. ; Nur, Erfan ; Konté, Kadère ; Afzali‐Hashemi, Liza ; Baas, Koen P. A. ; Schrantee, Anouk ; Wood, John C.

Abstract:

Sickle cell disease (SCD) is complicated by silent cerebral infarcts (SCIs), for which anemia is an important risk factor. Despite normal oxygen delivery (OD), cerebral vascular reserve (CVR), and cerebral metabolic rate of oxygen (CMRO2) are diminished in SCD, possibly causing the formation of SCIs. Voxelotor inhibits polymerization by increasing the hemoglobin oxygen binding, ameliorating hemolytic anemia. Furthermore, anemia is related to cardiac complications. Our aims were to assess the effect of voxelotor on markers of cerebral perfusion, cerebral oxygen metabolism, and markers of cardiac stress in SCD patients. Cerebral hemodynamics and oxygen metabolism were measured with MRI before and after 3 months of voxelotor treatment (1500 mg/day) in 18 adults with SCD (HbSS/HbSβ0‐thalassemia). Hemoglobin levels significantly increased (p = .001) and markers of hemolysis decreased (p < .05). OD increased from 6.5 (IQR, 6.0–7.1) mL O2/100 g/min to 8.1 (IQR, 7.2–8.7) mL O2/100 g/min (p = .001). CBF and CVR did not change. CMRO2 decreased from 2.0 (IQR, 1.9–2.1) mL O2/100 g/min to 1.9 (IQR, 1.6–2.1) mL O2/100 g/min (p = .03). N‐terminal pro‐B type natriuretic peptide (NT‐proBNP) levels decreased (p = .048) and maximum tricuspid regurgitation flow velocity (TRVmax) normalized in all but one patient with increased TRVmax. Voxelotor treatment in patients with severe SCD did not decrease CBF despite increased Hb levels. Cerebral oxygen metabolism slightly decreased, despite raised OD, most likely due to drug‐induced increase in oxygen binding. Nonetheless, voxelotor improved clinically validated markers of cardiac stress.

234

项与伏塞洛托相关的新闻（医药）

2025-03-24

·BiG生物创新社

天价基因疗法，狂热追捧中踩下急刹

作者｜与安 2025年3月，罗氏制药（Roche）宣布对旗下基因疗法子公司Spark Therapeutics启动“根本性重组”，全额减记24亿美元商誉。财务数据显示，罗氏已累计为 Spark 投入超10亿美元的重组成本，其中包括2024 年1.84亿美元的前期重组费用和2025年预计达3.4亿美元的追加支出。然而，这场持续六年的资本实验最终却惨淡收场，成为基因疗法领域最惨痛的收购案例之一。而这并非个例。过去三个月，基因治疗领域在希望与阵痛中交织前行，此前昭示熊熊野心的各家药企中，不乏惨淡收场的身影。跨国药企的管线重组、战略收缩与突破转型，再次将这一赛道的复杂性与不确定性推至台前，同时也代表着曾受资本狂热吹捧的基因疗法领域正逐步向理性发展回归转折。 01 43亿美元的深刻教训技术瓶颈下散场的资本盛宴 2019年，罗氏以43亿美元天价收购Spark Therapeutics，看中的是其全球首个获批的遗传性视网膜病变疗法Luxturna，以及血友病A基因疗法SPK-8011（dirloctocogene samoparvovec）的3期临床潜力。彼时Spark市值仅22亿美元，而罗氏以溢价125%的代价收购，创下当时基因疗法领域最大并购案，折射出行业对基因疗法商业化的狂热预期。然而截至 2024 年，Luxturna 年销售额仅 2050 万美元，同比暴跌 59%.此外，SPK-8011 项目则因安全性问题被紧急叫停，不达预期的商业表现和无法突破的技术难题，直接让这项大手笔并购案从"战略收购" 降级为 "产品交易"。迅速击穿资本泡沫的主要是三重瓶颈。首先是载体免疫性难题。Luxturna依赖的AAV2血清型载体，暴露了基因疗法最顽固的技术瓶颈之一，即预存免疫与后天免疫的双重绞杀。全球约30%-60%人群因自然感染携带AAV中和抗体，导致Luxturna治疗前必须进行抗体筛查。但Spark采用的"血清型切换"策略并未突破根本限制，数据显示超60%患者治疗后仍产生新中和抗体，直接导致疗效持久性不足。而后天免疫的影响在于，即使患者通过预存免疫筛查，治疗后仍可能因免疫系统激活产生新的抗AAV2免疫反应。其次是产能不足。Spark费城工厂病毒载体年产能仅5000剂，在适应症人群本就狭窄（全球约6000名患者）的情况下，覆盖率仍旧不足85%。患者等待期过长，市场渗透未达预期。最后则是临床表现的滑铁卢，完成收购时被列为重点的SPK-8011因安全性问题被叫停，于去年年底宣布终止研究。当病毒载体的免疫原性、生产工艺、长期安全性三大技术关卡未获突破时，资本堆砌反而加速了泡沫破裂。 02 火热势头下的行业寒冬难以走出的商业化困境与此同时，罗氏的受挫并非个例。从去年开始，基因治疗领域接连出现“利空”消息，整体成绩远低于预期，市场表现陷入疲软，多家行业明星宣布因进展不佳关停管线或业务重组。 2月21日，辉瑞宣布将停止进一步开发和商业化血友病B基因疗法Beqvez（SPK-9001）。这款药物于2024年4月刚获FDA批准，定价高达350万美元（约合人民币2537万元），在2024年全美最贵药物前十排行榜中，仅次于定价 425 万美元的 Lenmeldy，并列第二。 2024年全美最贵十款药物而Beqvez从获批上市到决定撤回，还不满一年时间。辉瑞表示，如此决定是因为“患者及医生对血友病基因疗法表现出的兴趣有限”，上市至今，尚未有患者接受过该药治疗。与此同时，辉瑞彻底清空了基因疗法管线。随着Beqvez退出，该司已没有任何基因疗法处于临床或商业化阶段。实际上，彼时将基因治疗领域列为“优先发展领域”的辉瑞，近三年来正在逐步撤离基因治疗领域。2023年7月，该司将早期研发阶段的基因疗法打包以高达10亿美元的价格卖给了阿斯利康旗下的罕见病部门Alexion；2024年6月，其针对杜氏肌营养不良症（DMD）的基因疗法fordadistrogene movaparvovec因CIFFREO Ⅲ期试验结果未达主要终点，陷入停滞；而到9月，更是撤回了市场上所有批次的镰状细胞贫血疗法Oxbryta，同时终止所有相关试验。因其临床数据表明，该药在镰状细胞患者群体中的总体效益不再足以覆盖其风险。至年底，辉瑞则终止了与Sangamo Therapeutics的全球合作及许可协议，放弃共同开发治疗A型血友病的基因疗法候选药物giroctocogene fitelparvovec（Giro-vec），尽管该药在去年完成的AFFINE III期临床试验中已取得积极结果，并已准备向FDA和EMA提交监管申请。临时放弃的主要原因大致有二，一是BioMarin同适应症的基因疗法Roctavian商业化进展不佳，市场表现惨淡，可谓现成的前车之鉴；二是辉瑞血友病新型抗体疗法Marstacimab于不久前才获批上市，作为给药便利性更加的抗体，对于基因疗法的市场空间也造成了一定冲击。总得来说，就是彼时看来，这款药物似乎难以实现大卖，而三个月后折戟的Beqvez也确实证实了这一点。而就在辉瑞宣布从基因疗法退场的前一天，曾经估值百亿的蓝鸟生物宣布卖身私募资金，一同踩下急刹。 2018年初，凭借β地中海贫血疗法Zynteglo（定价280万美元）、脑肾上腺脑白质营养不良疗法Skysona（定价300万美元）和镰状细胞病疗法Lyfgenia（定价310万美元），蓝鸟生物一时声名鹊起，股价飙升至每股2300美元，市值突破300亿美元。其“一次性治愈”叙事吸引了黑石、富达等顶级资本加持，成为首个基因疗法领域“十角兽”企业。然而，高昂定价和狭窄的患者群体让其商业化之路变得十分困难。受限于保险公司等支付方在高昂定价下的报销审批挑战，三款核心产品2023年总销售额为2910万美元，至今未实现盈利，其中，Zynteglo 2023年销售额为1670万美元，截至2024年11月累计治疗35例患者；Lyfgenia则仅完成4例患者治疗，虽与Vertex的CRISPR疗法同日获批，却因价格高出90万美元且因存在安全问题遭FDA发出黑框警告，市场份额遭到挤压。去年10月，蓝鸟宣布裁员25%并启动重组，但现金流缺口仍无法填补。最终，该司以2900万美元（不足巅峰市值0.1%）被私募基金凯雷/SK资本收购，对赌条款上限仅1亿美元，蓝鸟最终“断翅”。 03 市场洗牌中的希望与曙光大额盈利并非不可能一度受挫的基因疗法领域也并非毫无商业进展。与前文几款药物相比，Sarepta Therapeutics的Elevidys表现似乎截然不同。这是全球首款DMD基因疗法，可对携带任何类型DMD致病基因变异的患者生效，很好地填补了临床上的空白。值得注意的是，这还是和罗氏联合开发的疗法，同样高成本、高定价，售价320万美元，位列全美最贵药物第四（见图1）。2023年6月获FDA批准，8月正式上市销售，2024年便首度扭亏为盈。 2024年6月，Serepta扩大了该药的适应症，从原来的仅限于 4～5 岁具有行走能力的儿童，扩展为 4 岁及以上的儿童（无论是否有行走能力），进一步拓宽了市场覆盖面。至第三季度，Sarepta实现了净利润同比增长113%的好成绩，一举达到7619万美元。而诺华在AAV基因疗法上的投入则更是成效亮眼，针对脊髓性肌萎缩症（SMA）的Zolgensma可谓销冠，据其财报显示，2024年的年销售额仍然取得了12.14亿美元的好成绩。 Zolgensma2024年净销售额或许还有一大关键原因在于适应症的选择。反观让多位巨头或挣扎前行、或认栽止损的血友病基因疗法，市场表现不佳主要还是因为有凝血因子替代疗法和双抗药物可供患者选择。自身售价不菲，疗效也并无绝对优势，在可替代方案充足的情况下，难以打开市场的情况或许也并非难以预料。然而罗氏并未完全放弃。一方面，Spark的剩余业务将整合至集团，重组不影响Luxturna的持续供应，费城创新中心的一部分研发职能也仍然予以保留；另一方面，通过与Dyno Therapeutics达成的10亿美元合作，利用后者人工智能平台开发神经疾病新型AAV载体，罗氏将转向开发针对神经疾病的下一代基因疗法。基因疗法的故事远未终章。尽管挑战重重，部分企业仍以战略调整与技术升级寻找生机。2025年的春天，基因治疗领域仍在冰火之间跋涉。但随着临床技术的进步、监管政策的完善和支付体系的创新，当基因治疗的商业化摆脱纯资本驱动，构建起“技术-临床-支付”的完整闭环，这一领域仍然有望在迎来更为明朗开阔的前景，重唱高歌，再次高速前行。参考文献 https://www.fiercepharma.com/pharma/roche-overhauls-spark-gene-therapy-unit-recording-24b-full-impairment https://www.fiercepharma.com/pharma/pfizer-empties-gene-therapy-portfolio-discontinues-hemophilia-treatment-beqvez https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy https://www.pharmaceutical-technology.com/features/the-most-expensive-drugs-in-the-us/ 公众号文章 BioSeedin柏思荟辉瑞“泪洒”基因疗法 https://news.bioon.com/article/98498658028f.html 各家药企官网新闻动态和财报数据共建Biomedical创新生态圈！如何加入BiG会员？

基因疗法并购临床3期

2025-03-14

·BioSpace

Pfizer’s Pipeline Pruning Ends Early-Stage STING Trial

iStock, tomozina Pfizer was studying PF-07820435, an orally available agonist of the STING protein, for solid tumors. Pfizer has terminated a Phase I trial of its investigational STING therapy in advanced solid tumors, according to an update to the study’s clinicaltrials.gov page on Thursday. The move to discontinue the trial of PF-07820435 was based on “strategic business reasons,” according to the webpage. “The decision was not based on any safety and/or efficacy concerns.” BioSpace has reached out to Pfizer to confirm the discontinuation and will update this space accordingly. The Phase I trial was testing PF-07820435, with or without the PD-1 inhibitor sasanlimab, in nine patients with confirmed diagnoses of solid tumors. The main objective of the study was to establish the safety of PF-07820435 and detect early signals of efficacy, as well as determine the drug’s recommended dose for further studies. The study was initiated in February last year. STING, which stands for “stimulator interferon genes,” is a protein that plays a central role in a patient’s anti-cancer immune response by activating type I interferons and other inflammatory players. An orally available drug, PF-07820435 belongs to a class of drugs called STING agonists, which work by triggering the STING pathway and promoting immune activity against cancer cells. As of this writing, PF-07820435 remains on the pharma’s pipeline page . If Pfizer does eventually decide to completely drop PF-07820435, it will be the latest casualty in the pharma’s push to trim its pipeline and refine its business strategy. In December 2024, Pfizer turned its back on Sangamo Therapeutics and its gene therapy candidate giroctocogene fitelparvovec, which the companies were previously developing for hemophilia A. Phase III data released in July 2024 showed that 84% of patients treated with giroctocogene fitelparvovec maintained factor VIII activity above 5%, and majority achieved factor VIII levels greater than 15% of normal. Pfizer and Sangamo were supposed to Biologics License Application for giroctocogene fitelparvovec this year. The termination of the companies’ partnership will take effect on April 21, after which the pharma will transition the hemophilia A program back to Sangamo. Last month, Pfizer announced it was pulling its hemophilia B gene therapy Beqvez from markets worldwide—a decision the company pinned on “the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.” Pfizer, in September 2024, likewise withdrew Oxbryta, an oral hemoglobin S polymerization inhibitor that won the FDA’s approval in 2019 for sickle cell disease. The drug has since been linked to a higher occurrence of complications and death.

临床1期基因疗法上市批准临床3期临床结果

2025-03-06

·EndPoints

Novo Nordisk’s rare disease ‘company within a company’ has an open goal in sickle cell

Novo Nordisk is known for making therapies for some of the world’s most prevalent conditions, but it’s also working on candidates for some of the least common. Though the company’s rare disease unit is active in hemophilia and endocrine disorders, one of its most interesting up-and-coming assets is intended for sickle cell disease. Ludovic Helfgott, Novo’s head of rare disease, told Endpoints News on Thursday that data from a late-stage trial of etavopivat in sickle cell should come next year. That readout will indicate if the drug can overcome the worrying precedents set by other sickle cell treatments and whether Novo’s $1.1 billion buyout of Forma , which developed etavopivat, was in fact a good move. The company is targeting a 50% cut in the rate of vaso-occlusive crises (VOCs) in etavopivat’s Phase 3 study, according to Helfgott. This is roughly in line with the 46% cut in VOCs that the pyruvate kinase activator demonstrated in Phase 2 . In a Dec. 10 note, Leerink analysts said etavopivat’s VOC reduction in the mid-stage trial narrowly met the bar for clinical meaningfulness. However, they also wrote that etavopivat may have an important place in the sickle cell market given the high unmet need after Pfizer pulled its sickle cell drug Oxbryta worldwide last year over safety concerns. Plus, Europe and the UK a year ago rescinded approval of Novartis’ sickle cell therapy Adakveo, citing a no longer favorable “benefit-risk balance.” Helfgott is clear that Oxbryta’s withdrawal has no read-through to etavopivat. “We have a complete different molecule,” he said. “From a safety perspective, what Pfizer decided to do has no implication on what we do. It’s a completely different mode of action.” From a public health perspective, however, he noted that “it’s never a good thing when a medicine is withdrawn.” Patients have fewer treatment options, and Helfgott also said that there has been “a bit of mistrust in medicines” among sickle cell patients in recent years “and having a medicine withdrawn, unfortunately, doesn’t really help that.” Even with the much-anticipated arrival of sickle cell gene therapies, uptake has been slow. Vertex Pharmaceuticals’ Casgevy is slowly taking off , though it’s not a viable therapy for most sickle cell patients, who live in developing countries and have no way of paying roughly $2 million for treatment. Then there’s bluebird bio’s Lyfgenia, which stalled commercially. The biotech was sold to investors last month for just $29 million upfront. If etavopivat’s Phase 3 data are good enough for approval, it could have a pretty clear run at the sickle cell market, particularly if it shows a benefit on a secondary endpoint. The trial aims to demonstrate that the therapy can reduce the organ damage from sickle cells’ inability to deliver oxygen to patient tissues. “If you can show that you can preserve the organs better, then you have something which, for me, is really clinically relevant,” Helfgott said. Novo is investing heavily in its rare disease unit, which Helfgott refers to as “a company within a company.” It brings in around 8% to 10% of Novo’s annual revenue but accounts for more than 14% of the pharma’s R&D spend. The products it is developing are at least partly a hedge against hard times ahead. “The bigger the obesity [market], the bigger the diabetes [market], the bigger you need to compensate when loss of exclusivity will happen,” Helfgott said. “It’s really a strategic play for Novo Nordisk to have the rare disease segment alongside the more prevalent diseases.”

临床3期基因疗法临床结果细胞疗法临床2期

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外链

KEGG	Wiki	ATC	Drug Bank
D11330	伏塞洛托	B03XA	DB14975

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
溶血性贫血	欧盟	Pfizer Europe MA EEIG	2022-02-14
溶血性贫血	冰岛	Pfizer Europe MA EEIG	2022-02-14
溶血性贫血	列支敦士登	Pfizer Europe MA EEIG	2022-02-14
溶血性贫血	挪威	Pfizer Europe MA EEIG	2022-02-14
镰状细胞血症	美国	Global Blood Therapeutics, Inc.	2019-11-25

未上市

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适应症	最高研发状态	国家/地区	公司	日期
溶血	临床2期	法国	Pfizer Inc.	2023-03-22
慢性肾病	临床2期	美国	Global Blood Therapeutics, Inc.	2021-03-16
半规管裂综合征	临床2期	英国	Pfizer Inc.	2020-01-09
缺氧	临床2期	美国	Global Blood Therapeutics, Inc.	2016-06-01
特发性肺纤维化	临床2期	美国	Global Blood Therapeutics, Inc.	2016-06-01
血红蛋白SC疾病	临床1期	英国	Global Blood Therapeutics, Inc.	2014-12-01

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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


ASH2024	N/A	镰状细胞血症	-	Voxelotor 1500 mg	鏇淵築選簾鏇獵糧廠鹹(蓋顧淵膚壓鏇範築餘顧) = 選鹹選築觸齋鏇鹹淵夢艱廠簾淵淵網齋選範衊 (繭鏇衊鑰積艱鹽糧淵選, [1.2 ~ 7.3]) 更多	-	2024-12-08
ASH2024	N/A	镰状细胞血症	-	Voxelotor 1500 mg	選範遞憲簾夢網構廠夢(簾鹽鹹艱構餘觸鑰餘顧) = 願膚鑰淵願網齋壓鏇衊膚選製蓋廠構廠鏇鹹製 (鹽鑰積蓋網膚網鏇窪獵, 6.6 ~ 7.9) 更多	-	2024-12-08
ASH2024	N/A	-	-	Voxelotor 1500 mg daily	遞構願鹽蓋窪顧鹽觸製(鑰膚獵網糧構憲蓋鹹製) = 繭獵願鹽鬱淵構艱獵壓鑰鏇網獵觸觸衊願願衊 (簾獵膚窪鹹繭選鑰鑰簾 ) 更多	-	2024-12-08
ASH2024	N/A	-	-	Hydroxyurea (HU)	遞構願鹽蓋窪顧鹽觸製(鑰膚獵網糧構憲蓋鹹製) = 廠窪窪醖遞積鑰衊鑰遞鑰鏇網獵觸觸衊願願衊 (簾獵膚窪鹹繭選鑰鑰簾 ) 更多	-	2024-12-08
NCT05289570 (Voxelotor, CTgov)	临床2期	急性肺损伤	3	Voxelotor	積範構夢艱構窪鏇網選(構築遞鑰顧鏇網願繭艱) = 膚顧顧餘窪繭製膚窪鑰簾窪衊憲蓋餘積繭襯艱 (繭築窪餘範獵齋範襯鏇, 0.215) 更多	-	2024-07-24
EHA2024	N/A	镰状细胞血症 HbSS \| HbSβ0-thalassemia \| Hb	-	Voxelotor 1500 mg QD	觸鏇糧廠簾廠夢願夢鹽(襯鹹蓋網鹽築築壓衊網) = CMRO2 were unaffected 糧壓選鏇繭願網齋窪憲 (餘鹽憲廠糧鬱構網範顧 )	积极	2024-05-14
NCT05228834 (CTgov)	临床3期	镰状细胞血症	1	Placebo	遞憲糧製窪廠網齋積獵(夢遞簾繭餘觸築獵觸襯) = 願網鏇範願壓顧製選壓廠醖構遞鹹膚衊夢顧繭 (艱鬱醖觸構鏇壓餘獵遞, 範築製範積鏇鏇餘醖鑰 ~ 鑰醖願觸憲築鹹觸醖願) 更多	-	2024-05-02
NCT05199766 (ASH2023)	临床2期	-	25	Voxelotor 1500mg	網餘壓範醖窪築夢廠鹹(夢鑰網遞顧鑰構鏇鑰鬱) = 獵獵膚淵憲廠築夢餘鬱製繭齋範鹹願願簾餘觸 (願顧淵蓋獵襯壓襯艱積 ) 更多	-	2023-12-11
NCT04218084 (HOPE-KIDS 2, ASH2023)	临床3期	镰状细胞血症	708	Voxelotor	築製積鬱製觸艱憲鹽願(鏇膚鹹鬱繭範繭衊艱構) = 齋鏇餘齋遞醖觸鑰淵夢壓觸遞蓋築糧膚鏇製構 (顧製製膚範願廠壓積壓 )	-	2023-12-11
ASH2023	N/A	镰状细胞血症	19	Voxelotor-treated patients	餘艱淵積蓋製壓獵鹹膚(餘獵淵衊壓淵鏇鏇獵餘) = 構簾鑰憲鬱獵鬱顧鏇遞廠築構餘襯鬱顧繭獵簾 (窪範選範鹹顧衊廠膚獵 )	-	2023-12-09
NCT04400487 (ActIVe, CTgov)	临床4期	镰状细胞血症	25	Voxelotor	壓憲獵顧選蓋簾觸鏇齋(鑰襯選獵夢鬱鏇襯網築) = 遞淵鹹衊窪襯願構衊繭觸襯築製憲齋壓齋糧糧 (製膚夢鹽製膚繭鏇鏇網, 458.45) 更多	-	2023-11-21