FDA加速批准阿斯利康Etcamah用于ESR1突变晚期乳腺癌并授权Guardant360 CDx伴随诊断

FDA News Release
FDA Grants Accelerated Approval to a New Breast Cancer Treatment
- For Immediate Release:
The U.S. Food and Drug Administration today expanded treatment options for adult patients with advanced breast cancer, reflecting the FDA’s commitment to advancing medical innovation and getting new treatments to patients who need them.
FDA granted accelerated approval to Etcamah (camizestrant) in combination with a CDK4/6 inhibitor (abemaciclib, palbociclib, or ribociclib) for the treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer upon detection of estrogen receptor-1 (ESR1) mutation during aromatase inhibitor and CDK 4/6 inhibitor therapy, based on an FDA-authorized test.
ESR1 mutations are acquired resistance mutations that a tumor may develop during treatment with an aromatase inhibitor, a type of endocrine therapy that is a common front-line treatment for locally advanced or metastatic breast cancer. At the diagnosis of HR-positive metastatic breast cancer, fewer than 5 percent of patients will have this tumor mutation. After disease progression on an aromatase inhibitor, nearly 40 percent of patients will have this tumor mutation.
“Women living with metastatic breast cancer face an uphill battle as their tumors continuously evolve to escape treatment. We owe them every weapon in our arsenal.” said Acting FDA Commissioner Kyle Diamantas, J.D. “Today’s approval delivers a win to these patients by granting them a targeted therapy designed specifically to overcome resistance, giving them more time before their disease progresses.”
The accelerated approval program allows for earlier approval of drugs that treat serious conditions, and fill an unmet medical need based on surrogate or intermediate endpoints. In this case, accelerated approval of Etcamah was granted based on how long patients lived without their disease worsening, measured from the point when the resistance mutation was first detected in the blood. Since it is not yet confirmed whether intervening at this point, rather than at the time of confirmed disease progression, translates into a clinically meaningful benefit, the FDA has required confirmatory studies to verify and describe clinical benefit.
“I commend both the FDA and the sponsor for their commitment to advancing cancer care and securing this accelerated approval,” said Angelo de Claro, M.D., director of the FDA’s Oncology Center of Excellence. “This marks the first FDA approval of a cancer therapy guided by the detection of a resistance mutation in circulating tumor DNA (ctDNA) before imaging tests show that the disease is progressing. But additional evidence is needed to confirm clinical benefit.”
ctDNA is made up of tiny pieces of tumor DNA released into the blood, which can allow for earlier molecular detection of resistance mutations.
To ensure precise patient matching, the FDA also authorized the Guardant360 CDx assay as a companion diagnostic device to identify patients with breast cancer with ESR1 mutations for treatment with camizestrant.
Efficacy was evaluated in a clinical trial to assess switching to Etcamah, an oral tablet, in combination with a CDK4/6 inhibitor versus continuing an aromatase inhibitor in combination with a CDK4/6 inhibitor. Estimated median progression-free survival was 16 months in the Etcamah and CDK4/6 inhibitor arm and 9.2 months in the aromatase inhibitor and CDK4/6 inhibitor arm.
The prescribing information includes a boxed warning for the risk of irregular heart rhythm when Etcamah is taken together with certain other medications, as well as warnings and precautions for an abnormally slow heart rate and potential harm to an unborn baby.
Full prescribing information for Etcamah will be posted on Drugs@FDA.
On April 30, 2026, the FDA convened the Oncologic Drugs Advisory Committee for this application.
The accelerated approval was granted to AstraZeneca.
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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.