Roche Holding AG’s Avastin failed to extend the survival of patients with deadly brain tumors in a study that found no advantage in using the drug as a first-line therapy against the cancer known as glioblastoma.
In separate findings, the $6-billion-a-year treatment helped prolong the lives of women with an aggressive form of cervical cancer. Results from both trials were presented yesterday at the American Society of Clinical Oncology meeting in Chicago.
Roche, based in Basel, Switzerland, is counting on new uses of the medicine to help it reach the company’s projected $7 billion in peak annual sales. Avastin is already sold for use against colon, lung and brain tumors. In November 2011, U.S. regulators revoked the drug’s approval for use in breast cancer because it failed to prolong life in patients with the disease.
In the glioblastoma study, “the bottom line is that it didn’t show any benefit as a treatment for newly diagnosed patients,” said Mark Gilbert, a neuro-oncologist at the University of Texas MD Anderson Cancer Center in Houston who led the study. “The survival benefit was not there.”
The government-sponsored study of 637 glioblastoma patients found that those who got Avastin along with chemotherapy lived a median of 15.7 months compared with 16.1 months for patients given chemotherapy alone. Patients who received Avastin also had a higher rate of side effects including hypertension, bleeding, and pulmonary embolism.
Roche fell 2.4 percent to 233.30 Swiss francs at 9:10 a.m. in Zurich. The stock has returned 61 percent in the past year including reinvested dividends, compared with a 36 percent return for the Bloomberg Europe Pharmaceutical Index.
Avastin is approved in the U.S. for glioblastoma that has returned after initial treatment, and Roche is seeking approval in Europe and Japan.
A fast-growing type of cancer, glioblastoma accounts for about 15 percent of all brain tumors, according to the American Association of Neurological Surgeons. Some 23,130 Americans will probably be diagnosed with brain and nervous system cancers this year, according to the National Cancer Institute.
In the U.S., sales of Avastin for glioblastoma total about $170 million a year, Daniel O’Day, head of Roche’s pharmaceutical division, told investors in New York in February.
Some of the apparent brain tumor shrinkage seen with Avastin may actually be due to the drug’s tendency to make blood vessels in the brain less leaky, said Gilbert. By preventing fluid from leaking out, Avastin may make brain MRI scans look better even when cancer cells are not dying, said Gilbert.
Nonetheless, most glioblastoma doctors suspect the drug has a direct anti-cancer effect in at least some patients, Gilbert said in a phone interview.
Because the trial allowed patients who were initially assigned to chemotherapy alone to start taking Avastin after the chemotherapy failed, the trial in effect ended up measuring whether it made any difference to give Avastin sooner rather than later, said Gilbert.
Because of the risk of additional side effects, Avastin “can be reserved as a later treatment for most patients,” said Gilbert in a statement.
Separately, Roche released final data from its own 921-patient study of Avastin as an initial glioblastoma treatment at the conference. That study also found no improvement in survival among patients who were treated first with Avastin. The drug did delay the time it took the disease to spread further to a median of 10.6 months from 6.2 months, one of the primary goals of the study.
Even if there isn’t a survival difference, “keeping a patient’s brain cancer under control is important,” said Sandra Horning, global head of clinical development for cancer drugs at Roche, in an interview. She said the company found no evidence in the study that Avastin led to misleading brain scan results.
Meanwhile, Avastin helped women with an aggressive type of cervical cancer live almost four months longer in a trial that may give patients a new option for treatment.
Women whose cervical cancer had spread lived an average of 17 months with Avastin plus chemotherapy, compared to 13.3 months with chemotherapy alone, in a 452-patient study also presented yesterday at the cancer meeting. The results were the first time a drug like Avastin, also known as bevacizumab, was shown to help women with a gynecologic cancer live longer, the researchers said.
“This is a major advance when it comes to cervical cancer,” said Jamie Bakkum-Gamez, an assistant professor of obstetrics-gynecology who led the portion of the trial conducted at the Rochester, Minnesota-based Mayo Clinic.
Metastatic, or spreading, cervical cancer isn’t a curable disease. Patients tend to be among the younger population of people with gynecologic cancer, women in their 30s, 40s and 50s, Bakkum-Gamez said.
“They’re women that may be mothers, still young with young families,” she said.
Current standard care is chemotherapy alone, and perhaps radiation therapy or surgery in some cases, with a goal of relieving symptoms in the absence of a cure, she said. Debate now may center on how to weigh the cost of Avastin with the additional months of life the drug may be able to confer, Bakkum-Gamez said.
The cost of a course of treatment with Avastin for cervical cancer in the U.S. would be about $78,700, Roche said in an e-mailed statement. The company said it will discuss the cervical cancer data with the Food and Drug Administration and other regulators.