This Phase 2 study provided the efficacy data for vismodegib that led to its approval by the U.S. Food and Drug Administration (FDA) on Jan. 30, 2012.
"As a result of a strong collaboration between the Valley institutions, clinical development of this new medication, from the first clinical trial to the FDA approval, was led by the Valley researchers," said Dr. Sekulic, M.D and Ph.D., whose study found that vismodegib shrank advanced basal cell carcinoma tumors in 43 percent of patients with locally advanced disease and in 30 percent of patients whose disease spread to other organs.
The drug blocks the Hedgehog signaling pathway and was approved for treatment of locally advanced and metastatic basal cell carcinomas. Additional research noted encouraging results for patients with inherited genetic susceptibility for development of large numbers of BCC tumors, known as basal cell nevus syndrome.
"The availability of vismodegib and similar medications will really change the way we treat the patients with advanced forms of BCC, who had very limited options to date. In addition, this drug will likely offer a life-changing treatment for patients with basal cell nevus syndrome. It is possible that the drug may also be of benefit in treatment of earlier stages of BCC, and this is currently being evaluated in several clinical trials," Dr. Sekulic said.
"It is a landmark day for patients with basal cell carcinoma and all those involved in their care," said the NEJM editorial. However the editors cautioned that additional study of Hedgehog pathway inhibitors like vismodegib warrant further study so a larger number of patients may benefit.
FDA approval in five years is a remarkable achievement because clinical trials typically progress through three phases and can take up to 15 years to successfully complete, according to Mark Slater, Ph.D., Vice President of
|Contact: Steve Yozwiak|
The Translational Genomics Research Institute