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FDA Approves First Breast Cancer Drug to Treat BRCA Gene Mutation

The National Cancer Institute at the National Institutes of Health estimates that more than 252,700 women will be diagnosed with breast cancer in just this year alone.

Of those affected, about 40,600 people will die of the disease, but a new drug may help bring those numbers down and give a fighting chance to those battling the most common form of cancer in the U.S.

The Food and Drug Administration approved AstraZeneca’s Lynparza,  on January 12, 2018 to help treat those impacted by breast cancer.

It is the first time ever that a drug has been approved to help treat patients with metastatic breast cancer who also have a BRCA gene mutation, according to an FDA press release.

“This class of drugs has been used to treat advanced, BRCA-mutated ovarian cancer and has now shown efficacy in treating certain types of BRCA-mutated breast cancer,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval demonstrates the current paradigm of developing drugs that target the underlying genetic causes of a cancer, often across cancer types.”

Susan M. Domchek, Executive Director of the Basser Center for BRCA at the Abramson Cancer Center of the University of Pennsylvania, said Lynparza expands treatment options.

“Patients diagnosed with BRCA-related metastatic breast cancer are often younger than other breast cancer patients, and their disease is often much more aggressive and difficult to treat,” Domchek said in an AstraZeneca news release on January 12, 2018. “While there is currently no cure for metastatic breast cancer, today’s approval offers a new, targeted option that may help to delay disease progression for those patients.


Approximately 20 to 25 percent of patients with hereditary breast cancers and as much as 10 percent of patients with any type of breast cancer have a BRCA mutation.

BRCA genes are involved in repairing damaged DNA and normally work to prevent tumor development. However, mutations in these genes may lead to certain cancers, including breast cancers.

Lynparza works by blocking the enzyme involved in repairing damaged DNA.

“By blocking this enzyme, DNA inside the cancerous cells with damaged BRCA genes may be less likely to be repaired, leading to cell death and possibly a slow-down or stoppage of tumor growth,” according to the news release.

The drug can help patients beyond breast cancer. Lynparza was first approved in 2014 to treat patients with BRCA-mutated advanced ovarian cancer who have received three or more treatments of chemotherapy. It can also be used for the maintenance treatment of patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer whose tumors have at least partially responded to chemotherapy, the release states.

Patients are selected for treatment with Lynparza based on a genetic test called the BRACAnalysis CDx, an approved companion diagnostic. The FDA expanded the approval of this test to include the detection of BRCA mutations in blood samples from breast cancer patients.

Side Effects of Lynparza

The safety and effectiveness of Lynparza were based on a randomized clinical trial of 302 patients with HER2-negative metastatic breast cancer with a germline BRCA mutation, according to a news release.

The trial measured the length of time tumors did not have significant growth after treatment, also called “progression-free survival,” for patients taking chemotherapy only and compared it to patients taking Lynparza. The results showed the median progression-free survival for chemotherapy only was about 4.2 months, while it was 7 months for patients taking the new drug Lynparza, reducing the risk of disease progression or death by 42 percent, according to an AstraZeneca news release.

However, patients did experience side effects from Lynparza. Common side effects include anemia, nausea, fatigue, vomiting, common cold, respiratory tract infection, influenza, diarrhea, joint pain, headache and decreased appetite. More severe side effects include the development of blood or bone marrow cancers, inflammation in the lungs.

The most common side effect in the trial was nausea, reported by 58 percent of patients.  

The drug can also harm a developing fetus. The FDA advises that women should use effective contraception while taking this medication and avoid breastfeeding as it can harm a newborn.

Priority Review

The application was granted Priority Review, which allowed the FDA to take action on the application within 6 months as opposed to the 10 months of a standard review. The “priority review” designation is given to drugs that would significantly improve the safety or effectiveness of treatment, diagnosis or prevention of serious conditions when compared to standard applications, according to the FDA website.

“Designation of a drug as ‘Priority’ does not alter the scientific/medical standard for approval or the quality of evidence necessary,” the FDA states.

The designation also does not affect the length of the clinical trial period. Rather, it allows the FDA to direct more attention and resources to the drug’s evaluation.

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