Northwest Biotherapeutics Inc has been awarded special marketing authorization for its experimental brain cancer drug in Germany even before it has completed its late-stage trial. The company’s shares have increased in value as a result.
The company received a special hospital exemption in Germany that allows it to sell the injectable drug for five years, as well as the right to seek renewal after the exemption’s expiration. Northwest Bio CEO Linda Powers said the company has not yet requested or received official marketing approval for their potential blockbuster drug DCXav-L when the exemption was granted. DCXav-L belongs to a new class of drugs that recruits the body’s own immune system to target and destroy cancer cells. DCXav-L in particular harnesses dendritic cells including T-cells and B-cells.
Regulators awarded the special exemption to the company because of DCXav-L’s apparent advantages over standard treatments which offer little help to patients. CEO Powers said, “They realize we're still in our Phase III trial, but appear satisfied with the underlying biology of DCVax-L and the glimpses of effectiveness that were seen in early-stage trials.” Patients who used DCXav-L in small informal trials lived two to five times as long as patients who used standard treatments consisting of brain surgery, radiation, and treatment with Merck’s Temodar chemotherapy.
Standard treatment added two to five months of survival, allowing average patients to survive 14.5 months. In contrast, using DCXav-L plus standard treatment in the small Phase I/II trial lengthened survival of patients for three years, with no additional side effects. However, the study involved only 20 patients with newly diagnosed Glioblastoma multiforme (GBM) and so was not considered statistically significant due to its informal nature and small size.
The drug could potentially be worth over a billion dollars in annual sales if it wins full marketing authorization in Europe and U.S., according to industry analysts. At least 12,000 patients develop GBM in the U.S. alone every year.
Complete data from the formal late-stage trial of DCXav-L is expected to be available in 2015. The study is being conducted in Europe and the U.S. and involves 312 patients who were newly diagnosed with GBM and has undergone standard therapies. “If we meet the goals of the trial, we would plan to apply for marketing approval in Europe and the United States,” CEO Powers said.