2026-05-14 10:04 AM UTC / 路透社
作者：迪娜·比斯利与南希·拉皮德
2026年5月14日 10:04 AM UTC 更新于56分钟前
路透社/安德鲁·凯利/资料照片
- FDA批准扩大使用革命医药公司的试验性胰腺癌药物
- 肿瘤学家面临后勤障碍和资源紧张，需为患者纳入早期准入项目
- 该药物靶向90%胰腺癌患者体内存在的突变
5月14日（路透社）——美国癌症治疗中心正争相将患者纳入革命医药公司一款极具前景的胰腺癌药物早期准入项目，同时他们期盼着美国食品药品监督管理局能快速批准该药物。
美国食品药品监督管理局于5月1日批准了这项扩大使用项目，距革命医药公司宣布其每日一次的口服药物达拉克沙替尼（daraxonrasib）在晚期胰腺癌患者临床试验中使生存期翻倍还不到三周。胰腺癌是致死率最高的癌症之一，其5年生存率在所有癌症中处于最低水平。
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该公司曾向FDA申请，允许将这款药物免费提供给已接受过治疗、且癌症已扩散至身体其他部位的胰腺癌患者。
“公众得知了FDA的公告……这引发了大量患者的申请，”北韦尔健康系统扎克伯格癌症中心的肿瘤内科医生丹尼尔·金博士说道，“各癌症中心都在摸索如何与本院对接，开放项目规程以提供用药途径。”
肿瘤学家表示，获批并管理扩大使用项目需要时间，且癌症中心需在常规运营之外投入大量资源。
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这款药物是去年FDA采用新的快速审批程序后首批受理的产品之一，在完整申请提交后，有望在一两个月内获得批准。
总部位于加州红木城的革命医药公司未立即回应置评请求。
该公司首席执行官马克·戈德史密斯上周在一场电话会议中拒绝估算寻求早期用药的患者人数，也未透露完整FDA申报的时间框架。他表示：“我们正全力以赴推进此事。”
美国前内布拉斯加州参议员本·萨斯近日透露自己已处于胰腺癌4期，并在CBS新闻节目《60分钟》中表示自己正在服用该革命医药公司的药物。
可进一步开发的突破性疗法
这款药物靶向约90%胰腺癌患者体内存在的基因突变。临床试验数据显示，其可将患者中位生存期延长至13.2个月，而接受化疗的患者中位生存期仅为6.7个月。
“与现有最佳化疗方案相比，生存期翻倍是一项重大突破，”哥伦比亚大学/纽约长老会医院胰腺中心联合主任古拉姆·曼吉博士说道，“它不能治愈癌症，但我认为这款药物是我们可以在此基础上继续开发的全新突破。”
曼吉博士在其10年从医生涯中，仅为另一位患者申请过同情用药。他表示，近日在诊所当天就有7名患者向他咨询达拉克沙替尼的治疗方案。
他和其他癌症专家指出，让患者用上这款药物并不像开具FDA已批准药物的处方那么简单。
“患者早已看到了新闻稿，纷纷打来电话咨询，”希望之城医疗中心胰腺癌专家文森特·钟博士说道，“现在的挑战是如何推进这项工作。”
钟博士表示，该项目要求医生为每位患者向革命医药公司提交申请，若公司认定患者符合条件，还需将所有相关细节提交给FDA。医院监督委员会将对患者进行跟进。
“考虑到申请量，我不确定FDA那边会如何应对。我确信他们不希望一下子收到一万份申请，”钟博士补充道，该机构或许会转而制定更通用的入组规程。
曼吉博士表示，据他了解，癌症中心无需为扩大使用项目下的患者收集详细数据，但需报告严重不良反应或其他问题。
FDA未回应置评请求。这项快速审批券项目曾被马丁·马卡里博士誉为标志性成就，他于周二辞去FDA局长一职，此前他与特朗普政府官员已有数周的争执。
据美国癌症协会数据，今年美国约有6.7万人将被诊断为胰腺癌，其中5.3万人将死于该病。
“我们这么做是为了给患者提供帮助，”钟博士说道，“当然，我希望FDA能够审查相关数据，并且比常规审批更快地批准这款药物。”
本报由洛杉矶的迪娜·比斯利、图森的南希·拉皮德报道；芝加哥的朱莉·斯廷胡伊森补充报道；卡罗琳·胡默尔编辑
US cancer clinics scramble to get experimental Revolution Medicines pancreatic cancer drug
2026-05-14 10:04 AM UTC / Reuters
By Deena Beasley and Nancy Lapid
May 14, 2026 10:04 AM UTC Updated 56 mins ago
Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo
- FDA allows expanded access to Revolution Medicines’ experimental pancreatic cancer drug
- Oncologists face logistical hurdles and resource strain to enroll patients in early access program
- Drug targets mutation that occurs in 90% of pancreatic cancers
May 14 (Reuters) – U.S. cancer centers are scrambling to enroll patients in an early access program for a highly promising pancreatic cancer drug from Revolution Medicines while they await what they hope will be a speedy FDA approval.
The Food and Drug Administration allowed the expanded access program on May 1, less than three weeks after Revolution said the once-daily pill, daraxonrasib, doubled survival in a clinical trial of patients with advanced pancreatic cancer, among the deadliest of cancers with one of the lowest 5-year survival rates.
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The company had asked the FDA for permission to make it available free-of-charge for patients with previously treated pancreatic cancer that has spread to other parts of the body.
“The public caught wind of the FDA announcement… which has triggered a deluge of patient requests,” said Dr. Daniel King, medical oncologist at the Zuckerberg Cancer Center of Northwell Health. “Cancer centers are all figuring out how to engage with our own institutions, opening up the protocols to provide access.”
Oncologists said getting approval and managing the expanded access program will take time and will require cancer centers to dedicate substantial resources outside of their usual operations.
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The drug was one of the first products accepted by the FDA last year for its new expedited review process, and could potentially get approval a month or two after a complete application is filed.
Revolution Medicines, based in Redwood City, California, did not immediately respond to a request for comment.
CEO Mark Goldsmith, speaking on a conference call last week, declined to estimate how many patients would seek early access to the drug and did not give a time frame for a full FDA submission. “There’s a full-throttle effort to do it,” he said.
Former Nebraska U.S. Senator Ben Sasse recently revealed that he has Stage 4 pancreatic cancer and told the CBS news program “60 Minutes” that he is taking the Revolution drug.
A BREAKTHROUGH TO BUILD ON
The drug, which targets a genetic mutation found in about 90% of pancreatic cancers, was shown in a clinical trial to extend median survival to 13.2 months compared with 6.7 months for patients on chemotherapy.
“Doubling survival compared to best available chemotherapy is a big deal,” said Dr. Gulam Manji, co-director of the pancreas center at Columbia/New York-Presbyterian. “It is not a cure, but I think that this drug is a new breakthrough we can build on.”
In a 10-year career, Manji could recall seeking compassionate use of an experimental drug for just one other patient. On a recent day in the clinic, the Columbia oncologist said seven patients asked him about starting treatment with daraxonrasib.
Getting them access is not as simple as writing a prescription for an FDA-approved drug, he and other cancer specialists said.
“Patients are already aware of the press release and are already calling,” said Dr. Vincent Chung, pancreas cancer specialist at City of Hope. “The challenge now is how to proceed.”
The program requires physicians to submit requests for each individual patient to Revolution Medicines, Chung said, and if the company decides they are a good candidate, all those details then need to be submitted to the FDA. Hospital monitoring boards will need to follow the patients.
“Given the volume, I am not sure what will happen on the FDA side. I’m sure they don’t want to have 10,000 applications at once,” Chung said, adding that the agency may instead set up a more general enrollment protocol.
Manji said his understanding is that cancer centers will not be required to collect detailed data on patients treated under the expanded access program, but will need to report serious side effects or other issues.
The FDA did not respond to a request for comment. The expedited voucher program was touted as a signature achievement by Dr. Marty Makary, who resigned as FDA Commissioner on Tuesday after weeks of clashes with Trump administration officials.
Around 67,000 people in the United States will be diagnosed with pancreatic cancer this year, and 53,000 will die of the disease, according to the American Cancer Society.
“We are doing this as a service to our patients,” Chung said. “I’m hoping of course that the FDA is going to review the data and then there is an approval much sooner than is typical.”
Reporting By Deena Beasley in Los Angeles and Nancy Lapid in Tucson; additional reporting by Julie Steenhuysen in Chicago; editing by Caroline Humer and
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