When Roche tried to gain an approval to use T-DM1 against breast cancer based on mid-stage results from a single-arm study, the FDA sent its regulatory team packing--refusing to even review the application. But that experience hasn't stopped the pharma giant's Genentech subsidiary from taking a run at an approval of an experimental treatment for basal cell carcinoma, vismodegib, with a very familiar data package.
Vismodegib, also known as GDC-0449, has been advanced under a collaboration with Curis ($CRIS). The treatment registered a promising response rate in Phase II, according to researchers involved in the study. The treatment, which inhibits signaling in the Hedgehog pathway, was tested in one cohort suffering from locally advanced basal cell carcinoma and a separate cohort of metastatic patients.
In the mid-stage study, which involved 104 patients, vismodegib demonstrated an ability to shrink tumors or heal visible lesions in 43% of the patients with locally advanced BCC and 30% of patients with metastatic BCC. The median progression-free survival rate for both groups was 9.5 months.
Roche has a big edge in making its case for this drug. There is no approved therapy for basal cell carcinoma and the disease grows increasingly hard to stop as it advances. With no other options available, regulators may be willing to take their chances on vismodegib, which would set up a launch in 2012.
Shares of Curis--which stands to gain an $8 million payday if the application is accepted for review--jumped a bit more than 6% this morning. And it's hopeful that the hot wave of targeted cancer drugs will help sweep Roche's regulatory efforts to an approval.
"We believe that vismodegib is an excellent example of a targeted cancer drug that has been developed based on an understanding of the underlying molecular biology that drives the disease," Curis CEO Dan Passeri said in a statement. "We believe that this molecule has the potential to provide an important treatment option for patients with advanced BCC."
- check out the Roche release
- read the story from the Boston Business Journal
- here's the report from Bloomberg
Special Report: GDC-0449 - 10 promising late-stage cancer drugs