美国食品药品监督管理局批准百时美药物使其成为几十年来第一种新型精神分裂症药物-动脉网

FDA Approval of Bristol Myers Drug Makes It the First Novel Schizophrenia Med in Decades

MedCity News 等信源发布 2024-09-28 06:19

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Schizophrenia is currently treated with older medications with limited efficacy and troublesome side effects that lead many patients to stop taking them. The FDA has approved a novel Bristol Myers Squibb drug that takes a different approach to schizophrenia, introducing the first novel medication for the disorder in decades..

精神分裂症目前使用较老的药物治疗，疗效有限，副作用麻烦，导致许多患者停止服用。FDA批准了一种新型的百时美施贵宝药物，该药物对精神分裂症采取了不同的治疗方法，推出了数十年来第一种治疗精神分裂症的新型药物。。

For patients, approval of the drug, Cobenfy, offers a new treatment option with better tolerability. For BMS, the late Thursday regulatory decision marks a payoff for its multi-billion dollar acquisition of the drug’s developer as the pharmaceutical giant looks for new medicines with blockbuster potential to offset the loss of patent protection facing key products.

对于患者来说，批准该药物Cobenfy提供了一种新的治疗选择，具有更好的耐受性。对于BMS来说，周四晚些时候的监管决定标志着其以数十亿美元收购该药物开发商的回报，因为这家制药巨头正在寻找具有巨大潜力的新药，以抵消关键产品面临的专利保护损失。

BMS expects Cobenfy will become available in October..

BMS预计Cobenfy将于10月上市。。

The schizophrenia drugs currently available are antipsychotics that target and block dopamine receptors in the brain. The first generation of these drugs date to the 1950s and their side effects include movement disorders. Second-generation antipsychotics that emerged in the 1980s also block dopamine pathways, but their side effects include sleepiness, low blood pressure, weight gain, and sexual dysfunction.

目前可用的精神分裂症药物是针对和阻断大脑多巴胺受体的抗精神病药物。这些药物的第一代可追溯到20世纪50年代，其副作用包括运动障碍。。

BMS estimates that schizophrenia affects 2.8 million people in the U.S. The company calculates that about 60% of these patients either do not adequately improve or they experience intolerable side effects from currently available medications..

BMS估计，美国有280万人患有精神分裂症。该公司计算，这些患者中约有60%要么没有得到充分改善，要么经历了目前可用药物无法忍受的副作用。。

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Accelerating Claim Processing: Strategies to Shorten the Life of a Claim

加速索赔处理：缩短索赔寿命的策略

These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes.

这些战略和做法可以大大缩短索赔的生命周期，从而更快地解决问题，改善财务成果。

By Greenway Health

绿道健康

The BMS drug treats schizophrenia by blocking muscarinic cholinergic receptors. There are five types of muscarinic receptors found in the brain and some peripheral tissues. Biotech companies have been trying to develop drugs that specifically target and activate the M1 and M4 receptors without stimulating the other muscarinic receptors and causing side effects..

BMS药物通过阻断毒蕈碱胆碱能受体来治疗精神分裂症。在大脑和一些外周组织中发现了五种毒蕈碱受体。生物技术公司一直在尝试开发专门针对和激活M1和M4受体的药物，而不会刺激其他毒蕈碱受体并产生副作用。。

Cobenfy was developed in the labs of Karuna Therapeutics, where the drug was known in development as KarXT, shorthand for Karuna xanomeline-trospium chloride. Xanomeline, a small molecule that targets muscarinic receptors in the brain, was initially developed by Eli Lilly. While Lilly’s mid-stage tests showed efficacy in treating schizophrenia and Alzheimer’s disease-associated psychosis, results also showed side effects from the molecule’s targeting of receptors in peripheral tissue..

Cobenfy是在Karuna Therapeutics实验室开发的，该药物在开发过程中被称为KarXT，是Karuna xanomeline trospium chloride的缩写。Xanomeline是一种靶向大脑毒蕈碱受体的小分子，最初由礼来公司开发。虽然礼来公司的中期测试显示对治疗精神分裂症和阿尔茨海默病相关精神病有效，但结果也显示了该分子靶向外周组织受体的副作用。。

Karuna licensed xanomeline’s rights in 2012. The biotech’s innovation was pairing xanomeline with trospium chloride, a molecule that blocks muscarinic receptors — but only outside the brain and the central nervous system. This drug combination was designed to selectively target the M1 and M4 receptors in the CNS whose disrupted signaling is believed to contribute to psychosis and cognitive impairment.

卡鲁纳于2012年许可了xanomeline的权利。该生物技术的创新是将xanomeline与氯化特罗司匹姆配对，氯化特罗司匹姆是一种阻断毒蕈碱受体的分子，但仅限于大脑和中枢神经系统之外。该药物组合旨在选择性靶向中枢神经系统中的M1和M4受体，其信号传导被破坏被认为会导致精神病和认知障碍。

At the same time, this drug left alone the peripheral muscarinic receptors. When BMS reached a $14 billion deal to acquire Karuna last December, the schizophrenia drug was already under FDA review..

同时，这种药物使外周毒蕈碱受体单独存在。当BMS去年12月达成140亿美元收购卡鲁纳的协议时，这种精神分裂症药物已经在接受FDA的审查。。

FDA approval of Cobenfy was based on the results of two Phase 3 tests of the drug in adults. The identically designed placebo-controlled studies assessed patients using the Positive and Negative Syndrome Scale (PANSS), a rating scale that measures symptom severity in psychotic disorders such as schizophrenia.

FDA对Cobenfy的批准是基于该药物在成人中的两项3期试验结果。这项设计相同的安慰剂对照研究使用阳性和阴性综合征量表（PANSS）对患者进行评估，PANSS是一种衡量精神病性疾病（如精神分裂症）症状严重程度的评定量表。

Both trials showed that after five weeks, patients treated with the twice-daily capsule experienced statistically significant reductions in schizophrenia symptoms compared to placebo, achieving the main study goal..

两项试验均显示，与安慰剂组相比，服用每日两次胶囊的患者在五周后精神分裂症症状有统计学意义上的显着减轻，达到了主要研究目标。。

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What Healthcare Can Learn from Costco

好市多可以向医疗保健公司学习什么

The path forward is clear: employers must evaluate their health plan the same way they do their Saturday shopping excursion.

前进的道路是明确的：雇主必须像周六购物一样评估他们的健康计划。

By Jeff Bak, Imagine360 CEO and President

作者Jeff Bak，Imagine360首席执行官兼总裁

The most common side effects reported in the studies were gastrointestinal and included nausea, upset stomach, constipation, abdominal pain, vomiting, and diarrhea. Unlike the first- and second-generation antipsychotics, Cobenfy’s label does not carry a black box warning for adverse effects. The BMS drug’s label does include warnings for urinary retention, increased heart rate, gastric retention, and angioedema.

研究中报道的最常见的副作用是胃肠道反应，包括恶心，胃部不适，便秘，腹痛，呕吐和腹泻。与第一代和第二代抗精神病药物不同，Cobenfy的标签上没有关于不良反应的黑匣子警告。BMS药物的标签确实包括尿潴留，心率加快，胃潴留和血管性水肿的警告。

The label also recommends against use of the drug in patients with liver impairment..

标签还建议不要在肝功能损害患者中使用该药物。。

Summer Colling, a senior analyst at Citeline, said the BMS drug’s new mechanism of action marks a new era in psychiatric drug development. But she added that one drawback is the drug’s twice-daily administration, which is less convenient than available antipsychotics with once-daily dosing or even longer dosing schedules..

Citeline的高级分析师Summer Colling表示，BMS药物的新作用机制标志着精神病药物开发的新时代。但她补充说，该药物的一个缺点是每天服用两次，这比现有的抗精神病药物每天服用一次甚至更长的服用时间表更不方便。。

“KarXT has a strong efficacy profile, but it is difficult to compare PANSS reduction scores to marketed atypical antipsychotics without head-to-head trials,” Colling wrote in an email. “Having said that, statistically significant effects were observed as early as week two after KarXT treatment, which could be an important differentiating factor for the drug since current therapies take much longer, often three to four weeks, to show clinical improvement.”.

柯林斯在一封电子邮件中写道：“KarXT具有很强的疗效，但很难将PANSS降低评分与未经头对头试验的市售非典型抗精神病药物进行比较。”。“尽管如此，早在KarXT治疗后第二周就观察到了统计学上显着的效果，这可能是该药物的重要区别因素，因为目前的治疗需要更长的时间，通常需要三到四周才能显示临床改善。”。

Colling said it’s unlikely the BMS drug will be used as a first-line schizophrenia treatment due to cost and insurance policies. She expects it will be prescribed for patients who do not benefit from or cannot tolerate at least two generic antipsychotics, such as risperidone and olanzapine.

柯林说，由于成本和保险政策的原因，BMS药物不太可能被用作一线精神分裂症治疗。她预计，这将被处方用于那些不能从利培酮和奥氮平等至少两种非专利抗精神病药物中获益或不能耐受的患者。

BMS set a $1,850 per month wholesale price for Cobenfy, or more than $22,000 per year, which is in line with other brand name antipsychotics. Competition is coming from other muscarinic receptor-targeting drugs in development. The most advanced is emraclidine, which AbbVie added to its pipeline through the $8.7 billion Cerevel Therapeutics acquisition that closed in August.

BMS为Cobenfy设定了每月1850美元的批发价格，即每年超过22000美元，这与其他品牌的抗精神病药物一致。竞争来自正在开发的其他毒蕈碱受体靶向药物。最先进的是emraclidine，AbbVie通过8月份完成的价值87亿美元的Cerevel Therapeutics收购，将其添加到其管道中。

The once-daily drug is currently completing two Phase 2 studies designed to support a regulatory submission. In a note sent to investors on Friday, Leerink Partners analyst David Risinger said AbbVie expects to seek approval for emraclidine in the second half of 2025, potentially setting the stage for a regulatory decision in the first half of the following year.

这种每日一次的药物目前正在完成两项旨在支持监管提交的第二阶段研究。Leerink Partners分析师DavidRisinger在周五发给投资者的一份报告中表示，AbbVie预计将在2025年下半年寻求埃姆拉西丁的批准，这可能为明年上半年做出监管决定奠定基础。

He added that available data suggest AbbVie’s schizophrenia drug likely offers lower efficacy but better safety than Cobenfy, but the BMS drug will have about an 18-month head start..

他补充说，现有数据表明，AbbVie的精神分裂症药物可能比Cobenfy的疗效更低，但安全性更好，但BMS药物将领先约18个月。。

William Blair estimates Cobenfy could achieve peak sales of $2 billion in 2030 in schizophrenia alone. But in a Friday research note, analysts Matt Phipps and Myles Minter noted that pivotal tests are underway testing the drug in Alzheimer’s-associated psychosis and adjunctive schizophrenia. If Cobenfy wins approval in these indications, the drug’s sales yearly sales could reach between $3 billion and $5 billion..

威廉·布莱尔（WilliamBlair）估计，科本菲（Cobenfy）仅在精神分裂症方面的销售额就可能在2030年达到20亿美元的峰值。但在周五的一份研究报告中，分析师马特·菲普斯（MattPhipps）和迈尔斯·明特（MylesMinter）指出，目前正在进行关键测试，以测试该药物治疗阿尔茨海默病相关精神病和辅助性精神分裂症。如果Cobenfy在这些适应症方面获得批准，该药物的年销售额可能达到30亿至50亿美元。。

“We believe the first-mover advantage in this novel drug class for schizophrenia, meaningful enthusiasm around the availability of a new modality in schizophrenia, and favorable product profile for Cobenfy should support strong early adoption,” the William Blair analysts wrote.

威廉·布莱尔（WilliamBlair）的分析师写道：“我们认为，这种新型精神分裂症药物类别的先发优势，对精神分裂症新模式的可用性的有意义的热情，以及对Cobenfy有利的产品简介，都应该支持早期采用。”。

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