The Food and Drug Administration on Thursday

食品和药物管理局周四

approved an Alnylam Pharmaceuticals medicine

批准了Alnylam制药公司的一种药物

for a serious heart condition, a decision that should help the biotechnology firm secure its position as one of the sector’s most valuable companies.

由于一种严重的心脏病，这一决定应该有助于这家生物技术公司确保其作为该行业最有价值的公司之一的地位。

The agency cleared the drug, called Amvuttra, for people with a cardiac form of the rare disease transthyretin amyloidosis. Alnylam already sells the therapy for individuals with a genetic type of the disease that causes progressive nerve damage.

该机构批准了名为Amvuttra的药物，用于治疗患有罕见病转甲状腺素蛋白淀粉样变性的心脏病患者。Alnylam公司已经在销售针对导致进行性神经损伤的遗传类型疾病的疗法。

Transthyretin amyloidosis with cardiomyopathy is considered deadlier than the nerve type, and often leads to hospitalizations and heart failure. It’s also thought to be more common, making it a target for drugmakers seeking a lucrative market opportunity.

淀粉样蛋白心肌病被认为比神经型更致命，常导致住院和心力衰竭。它也被认为更为常见，因此成为制药商寻求高利润市场机会的目标。

In testing, treatment with Amvuttra led to a

在测试中，使用Amvuttra治疗导致了

28% lower risk of recurrent cardiovascular events or death from any cause

复发性心血管事件或任何原因导致的死亡风险降低28%

than placebo in people with the disease. The drug similarly reduced that risk versus placebo among people who weren’t receiving another medication for the condition, Pfizer’s tafamidis, at the study’s start.

与安慰剂相比，该药物在患病人群中的效果更佳。对于在研究开始时未接受另一种药物（辉瑞的tafamidis）治疗的患者，该药物同样降低了风险。

Both measures were the study’s main goals after

这两项措施都是研究的主要目标之后

statistical changes Alnylam made to the study

Alnylam 对研究进行的统计更改

a year ago.

一年前。

Amvuttra labeling specifies treatment can

Amvuttra标签说明治疗可以

reduce hospitalizations or death

减少住院或死亡

resulting from heart complications, as well as urgent visits for heart failure. That indication could help the drug’s commercial prospects, as tafamidis and the other available medication, BridgeBio Pharma’s Attruby, are also cleared for that use.

因心脏并发症以及心力衰竭的紧急就诊。这一适应症可能有助于该药物的商业前景，因为tafamidis和另一种可用药物BridgeBio Pharma的Attruby也获批用于该用途。

The approval provides a “new and clinically differentiated treatment option that has been shown to improve outcomes,” said CEO Yvonne Greenstreet, in a statement.

首席执行官伊冯·格林斯特里特在一份声明中表示，该批准提供了“一种新的、临床差异化的治疗选择，已被证明可以改善结果”。

Amvuttra will initially be priced in line with the cost in its other approved indication, for which a year’s supply has a roughly $476,000 list price, the company

Amvuttra的定价将与其在其他已批准适应症中的成本保持一致，一年的供应量标价大约为47.6万美元，该公司表示。

revealed on a conference call

在电话会议上透露

. Alnylam expects the drug’s net price to then gradually decrease as patient uptake climbs. By comparison, tafamidis has a list price of over $250,000 while Attruby’s yearly cost is about $244,000.

Alnylam预计，随着患者使用量的增加，该药物的净价格将逐渐下降。相比之下，tafamidis的标价超过25万美元，而Attruby的年费用约为24.4万美元。

Approval of Amvuttra in transthyretin amyloidosis, or ATTR, cardiomyopathy is important for the future of Alnylam, which pioneered a drugmaking method called RNA interference.

批准Amvuttra用于转甲状腺素蛋白淀粉样变性（ATTR）心肌病，对开创了RNA干扰药物制备方法的Alnylam公司的未来至关重要。

Though the company has developed four other marketed medicines — cementing itself as one of the industry’s most prolific developers — it’s still not profitable. Alnylam has accumulated a deficit of more than $7 billion since its 2002 founding, including $1.85 billion in combined net losses over the last three years, according to its .

尽管该公司已经开发了四种其他已上市的药物——巩固了其作为业内最多产的开发商之一的地位——但它仍然没有盈利。根据其数据显示，自2002年成立以来，Alnylam已累计亏损超过70亿美元，其中包括过去三年合计净亏损18.5亿美元。

most recent annual report

最近的年度报告

.

。

Wall Street analysts and investors view approval in ATTR cardiomyopathy as Alnylam’s best opportunity yet to turn a regular profit. Historically, the disease has been underdiagnosed as it can be mistaken for other conditions. But better awareness, diagnostic tools and the availability of tafamidis since 2019 have grown the market, leading analysts to forecast annual sales for drugs that treat the condition rising to $15 billion to $20 billion over time.

华尔街分析师和投资者认为，Alnylam在ATTR心肌病领域的获批是其迄今为止实现常规盈利的最佳机会。历史上，这种疾病由于可能被误诊为其他病症而一直存在诊断不足的情况。但随着认知的提高、诊断工具的改进以及自2019年以来tafamidis的上市，市场不断扩大，分析师预测治疗该疾病的药物年销售额最终将增长至150亿至200亿美元。

Global sales of tafamidis surpassed $5 billion last year..

去年，Tafamidis的全球销售额超过了50亿美元。

Alnylam has sought to prove its medicines can deliver powerful benefits by “silencing” the misfolded protein implicated in the disease rather than stabilizing it like tafamidis does. The company came close to an approval with an earlier drug, Onpattro. But the FDA

Alnylam试图证明其药物能够通过“沉默”与该疾病有关的错误折叠蛋白，而不是像tafamidis那样稳定它，从而带来显著的疗效。该公司早前的一种药物Onpattro曾接近获得批准。但是，美国食品药品监督管理局（FDA）…

rejected it in 2023

在2023年拒绝了它

after determining the supportive data weren’t meaningful enough to warrant an OK, and Alnylam abandoned seeking a clearance of Onpattro in cardiomyopathy.

在确定支持性数据不够有意义，不足以证明获得批准是合理的之后，Alnylam 放弃了寻求 Onpattro 在心肌病方面的许可。

The company tried again with Amvuttra, which works similarly to Onpattro but is administered through an under-the-skin injection rather than an infusion. Alnylam also ran the main trial supporting its approval for long enough to assess whether treatment could extend lives — the kind of evidence underlying tafamidis’ approval — and .

公司再次尝试了Amvuttra，它的作用与Onpattro相似，但通过皮下注射给药而不是输液。Alnylam还进行了支持其批准的主要试验，时间足够长以评估治疗是否能够延长生命——这是tafamidis获批所依据的那种证据。

detected a benefit

检测到一种益处

that appears to have satisfied U.S. regulators.

这似乎已经让美国监管机构满意。

Amvuttra’s sales potential may not end up being as high as some on Wall Street predict, however. The drug will compete for market share tafamidis and

然而，Amvuttra的销售潜力最终可能不会像华尔街一些人预测的那么高。该药物将与tafamidis竞争市场份额。

Attruby

属性

, both of which are taken orally. The latter was

，这两种药物都是口服的。后者是

approved last year

去年批准

and is off to a

并且开始了

faster-than-expected start

比预期更快的开始

. Alnylam’s drug wasn’t tested directly against either one and, while cross-trial comparisons can be misleading, its results don’t appear strong enough to indicate vutrisiran is clearly superior. Physicians interviewed by BioPharma Dive last year

Alnylam的药物并未直接与其中任何一种进行测试，而且，虽然跨试验比较可能具有误导性，但其结果似乎并不足以表明vutrisiran明显更优。BioPharma Dive去年采访的医生们也表示：

said

说

that deciding which drug to start new patients on, or switch to if one therapy stops working, will be difficult.

决定让新患者开始使用哪种药物，或者在一种疗法失效时改用哪种药物，将会很困难。

Given the drugs’ costs, insurers may not approve use of Amvuttra alongside tafamidis or Attruby. Generic versions of tafamidis, which could arrive later this decade, will further shift the market balance.

鉴于这些药物的成本，保险公司可能不会批准将Amvuttra与tafamidis或Attruby同时使用。预计将在本十年后期上市的tafamidis仿制药将进一步改变市场平衡。

Ionis Pharmaceuticals

艾奥尼斯制药公司

and

和

Intellia Therapeutics

Intellia 治疗公司

are advancing other medicines, meanwhile.

同时，其他药物也在不断进步。

In an interview ahead of the FDA’s decision, Chief Commercial Officer Tolga Tanguler said Amvuttra can help grow the market. While diagnosis rates have climbed since tafamidis’ initial approval, the majority of patients aren’t on treatment, leaving room for new entrants to gain share, he said.

在FDA决定之前的采访中，首席商务官托尔加·坦古勒表示，Amvuttra有助于市场增长。他说，自tafamidis最初获批以来，诊断率虽有所上升，但大多数患者仍未接受治疗，这为新进入者提供了获取份额的空间。

Tanguler said Alnylam sees Amvuttra as “well positioned to become the standard of care.” He noted how adherence to the drug, which is injected four times a year, is very high in the neuropathy form of ATTR. “You don’t get that on an oral therapy,” he added. Good results “can only happen if you're actually taking your medicine.”.

唐古勒表示，Alnylam认为Amvuttra“非常有望成为治疗标准”。他指出，在ATTR的神经病变形式中，这种每年注射四次的药物依从性非常高。“口服疗法是无法达到这种效果的，”他补充道。好的结果“只有在你真正服药的情况下才会发生”。

Alnylam’s therapy also was tested in people who were already on other background therapies, among them tafamidis, which “set the bar very high.” Amvuttra nevertheless succeeded, with benefit reported across different subgroups.

Alnylam的疗法还在已经接受其他背景疗法的患者中进行了测试，其中就包括“门槛非常高”的tafamidis。然而，Amvuttra依然取得了成功，并在不同的亚组中显示出益处。

'I have every confidence we'll be able to communicate that very effectively to both payers and prescribers,” Tanguler said.

“我完全相信，我们将能够非常有效地向支付方和开处方者传达这一信息，”Tanguler说。

The company projects Amvuttra and Onpattro will generate about $1.6 billion to $1.7 billion in combined revenue this year, versus just over $1.2 billion last year. Tanguler expects Amvuttra’s usage to grow over the second half of the year and accelerate after.

该公司预计，Amvuttra 和 Onpattro 今年将带来约 16 亿至 17 亿美元的总收入，而去年仅为 12 亿美元。Tanguler 预计 Amvuttra 的使用量将在今年下半年增长，并在此后加速。

Editor’s note: This story has been updated with Amvuttra’s list price.

编辑注：本文已更新了Amvuttra的定价信息。