Roche ($RHHBY) and Genentech won a landmark approval for the cancer drug vismodegib today. After gaining significant efficacy data from a Phase II study on basal cell carcinoma, the drug giant not only went straight to the regulators for a decision, they won the approval well ahead of the agency's decision deadline. And top regulators signaled that they would look kindly on other developers who take the same approach to targeted therapeutics.
"Our understanding of molecular pathways involved in cancer, such as the Hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "This approach is becoming more common and will potentially allow cancer drugs to be developed more quickly. This is important for patients who will have access to more effective therapies with potentially fewer side effects."
The treatment, which inhibits signaling in the Hedgehog pathway, was tested in one cohort suffering from locally advanced basal cell carcinoma and a separate cohort of metastatic patients. In the mid-stage study, which involved 104 patients, vismodegib demonstrated an ability to shrink tumors or heal visible lesions in 43% of the patients with locally advanced BCC and 30% of patients with metastatic BCC. The median progression-free survival rate for both groups was 9.5 months.
Vismodegib, which will be marketed as Erivedge, was developed in collaboration with Curis ($CRIS).
The approval may well help spur other developers to consider taking the same approach. But it won't be easy. Roche tried to get an approval for the impressive T-DM1 on mid-stage data, but the regulators wouldn't even consider that application. A big plus this time is the lack of any other treatments for these patients.
- here's the press release from the FDA