By Lisa Seachrist
WASHINGTON — In response to unanimous advisory panel support for the drug and in anticipation of marketing approval, the National Cancer Institute (NCI), in collaboration with Genentech Inc. and the FDA, will be offering all women enrolled in the expanded access lottery program access to treatment with Herceptin, Genentech's monoclonal antibody for the treatment of metastatic breast cancer.
The lottery was begun last February and managed by the NCI, in Bethesda, Md., as a means to provide access to the limited supplies of the experimental drug to patients who didn't meet the criteria for conventional research studies. NCI is expanding its studies with Herceptin to allow about 400 patients who didn't receive Herceptin by way of the lottery to be treated with the drug.
"This is a very new type of therapy that is likely to be very important for breast cancer patients," said Tom Davis, senior investigator with the NCI. "The amount of drug that Genentech has been able to manufacture before now has been quite limited. With the new expanded access program, we are prioritizing those who've not been selected in the lottery."
Herceptin is a humanized monoclonal antibody against the growth factor receptor HER2. About one-third of all breast cancers overexpress the HER2 gene. In such tumors, the cell surfaces are marked with receptors that fuel their growth. Rather than attacking cells directly, Herceptin neutralizes the receptors.
On Sept. 2, the FDA's Oncologic Drugs Advisory Committee voted 11-0 to recommend approval of Herceptin. With that recommendation, Genentech expects to receive approval for the drug before year's end. (See BioWorld Today, Sept. 3, 1998, p. 1.)
Genentech has been supplying the NCI with Herceptin when excess supply of the drug was available, said company spokesman Geoff Teeter. "We've provided drug for the expanded access program in February, July and now September," he said. "We partnered with NCI to get as wide a geographic availability for Herceptin as possible."
In May, shortly after submitting its biologics license application, the firm ramped up production of Herceptin. As a result, there is plenty in supply to treat the patients who were waiting for their numbers to come up in the lottery.
Patients Not In Lottery May Join TRC-9801
All patients who entered the lottery before 4:30 p.m. Sept. 18, but were not selected, will now be able to receive the drug by either participating in an ongoing trial, known as TRC-9801, being conducted at one of 30 NCI cancer centers or by entering a new clinical trial, known as TP-9801, that allows the patient's primary oncologist to deliver the drug. Both trials are open-label studies, so all patients receive active Herceptin.
In addition, if waiting for the commercial release of Herceptin isn't a reasonable option for them, patients who haven't entered the lottery yet may join the TRC-9801 trial being conducted at clinical centers.
"We are in discussions with the agency," Teeter said. "But the ball is now in their court and we have no way to know when Herceptin will be approved."
Nevertheless, Teeter said, "some women may be best served by waiting for the drug to be approved." Davis agreed, pointing out it takes two to three weeks from contacting the cancer center to be enrolled on the protocol and receive the drug. Should the cancer centers be deluged with requests to go on study, the delays could be even longer.
While the company wouldn't try to predict when Herceptin is likely to receive marketing clearance, the NCI's Davis told BioWorld Today that, based on the conversations he's had with the FDA, approval could come within the next two to four weeks.
Genentech's stock (NYSE:GNE) closed at $70.875, up $0.625. *