Roche has received accelerated approval for Avastin (bevacizumab) from the US Food and Drug Administration (FDA) for people with glioblastoma with progressive disease following prior therapy.
The new indication for Avastin was granted under the FDA's accelerated approval programme that allows provisional approval of medicines for cancer or other life-threatening diseases. It follows the unanimous vote by the FDA Oncologic Drugs Advisory Committee (ODAC) on March 31.
"People with this type of brain cancer have had no new treatments in more than a decade," said Timothy Cloughesy, M.D., director, Neuro-Oncology Program of the Jonsson Comprehensive Cancer Center at the University of California, Los Angeles. "After so many years with little progress in this field, Avastin was associated with a durable tumour response and doctors now have a new medicine to offer patients."
"Today's approval would not have been possible without the dedication of physicians, patient advocates, the FDA and most importantly the people who participated in the clinical trials and their families who had the courage to support them," said William M. Burns, CEO of Roche's Pharmaceuticals Division. "A global phase III trial in patients with newly diagnosed glioblastoma will soon begin enrolment to further evaluate Avastin in this setting."
The National Brain Tumor Society, an American patient group, has welcomed the FDA's accelerated approval of Avastin. "A brain cancer diagnosis is devastating because the tumours invade brain tissue and can cause rapid deterioration," said Harriet Patterson, director of patient services for the National Brain Tumor Society. "Until now, people with relapsed glioblastoma have had almost no treatment choices and little hope."
Following initial treatment with chemotherapy and radiation, more than 90 per cent of patients with glioblastoma will see their cancer return and there are few effective treatments when the initial therapy stops working. Median survival following progression of this cancer is approximately six months.
The effectiveness of Avastin for this aggressive form of brain cancer is based on an improvement in objective response rate from the BRAIN study (AVF3708g) and an NCI study (NCI 06-C-0064E). Currently, no data are available from randomized controlled trials demonstrating an improvement in disease-related symptoms or increased survival with Avastin in glioblastoma.
Roche has also filed an application with the EMEA in December 2008 for marketing authorisation for Avastin as a therapy for patients with previously treated glioblastoma. The BRAIN study forms the basis of both US and European regulatory submissions.
The accelerated approval is based on independently reviewed data from the BRAIN study which was an open-label, multicenter, non-comparative Phase II study that included 167 patients with glioblastoma that had progressed following initial treatment with temozolomide and radiation. Patients were randomized into two arms: Avastin alone or Avastin in combination with irinotecan. A primary endpoint of the study was objective response rate. Response was assessed by magnetic resonance imaging (MRI) and measured using World Health Organization radiographic criteria along with decreased or stable corticosteroid use. MRI does not necessarily distinguish between the tumor, swelling (edema), or tissue death (necrosis) caused by prior radiation therapy.
Glioma (cancer of the glial cells) is the most common type of malignant primary brain tumour (a tumour that originates in the brain), accounting for approximately one third of all cases diagnosed. Glioblastoma (or glioblastoma multiforme; GBM) is the most common and most aggressive type of glioma. The prognosis for patients with GBM is poor, and generally depends on the success of surgery to remove the tumour.
Glioblastoma affects approximately 10,000 people per year in the United States. Following initial treatment, glioblastoma tumours nearly always return and currently, there are limited treatment options for patients when these relapses occur. According to historical estimates, less than 10 per cent of patients with recurrent GBM respond to treatment and approximately 15 per cent will live six months without their disease getting worse.
Avastin is a biologic antibody designed to specifically inhibit the vascular endothelial growth factor (VEGF) protein that plays an important role in the development and maintenance of blood vessels, a process known as angiogenesis. VEGF is a potent activator of angiogenesis throughout the lifecycle of a tumour and is thought to be critical to a tumour's ability to grow and spread in the body (metastasize). Avastin is approved in EU for the treatment of the advanced stages of four common cancer types: colorectal cancer, breast cancer, lung cancer and kidney cancer. More than 500,000 patients have been treated with Avastin so far.