The US Food and Drug Administration (FDA) has accepted Roche's New Drug Application for vismodegib for the treatment of adults with advanced basal cell carcinoma (BCC) for whom surgery is considered inappropriate. The application has been granted Priority Review status and the FDA confirmed the action date is March 8, 2012.

Vismodegib is an investigational, oral, targeted medicine designed to selectively inhibit signalling in the Hedgehog pathway, which is implicated in more than 90 per cent of BCC cases. BCC is the most common type of skin cancer, which is generally considered curable by surgery. However, when it advances, BCC can cause disfiguring and debilitating effects and in some patients can ultimately be life-threatening. Currently, there are no effective treatment options for advanced BCC.

“We are pleased the FDA has granted priority review for vismodegib and we look forward to working with the Agency on the review of the data," said Hal Barron, M.D., chief medical officer and head, global product development. “We hope to provide people with the first FDA-approved medicine for this potentially disfiguring, and in some cases fatal, disease as soon as possible.”

The vismodegib application is based on results from the pivotal ERIVANCE BCC study. The trial showed vismodegib substantially shrank tumours or healed visible lesions (objective response rate, or ORR) in 43 per cent of patients with locally advanced BCC (laBCC) and 30 percent of patients with metastatic BCC (mBCC), as assessed by independent review, the primary endpoint of the study. The ORR as assessed by study investigators, a secondary endpoint, was 60 per cent for laBCC and 46 per cent for mBCC. The median progression-free survival (PFS) by independent review for both metastatic and locally advanced BCC patients was 9.5 months.

BCC is the most common type of skin cancer in Europe1, Australia1,2, and the USA3. The disease is generally considered curable if the cancer is restricted to a small area of the skin. However, in a very small group of people, if the disease is left untreated or recurs after surgery, it becomes locally advanced, and the cancer may invade further into surrounding tissues such as sensory organs (ears, nose and eyes), bones or other tissues.

Vismodegib is an investigational medicine designed to selectively inhibit abnormal signaling in the Hedgehog pathway, which is an underlying molecular driver of BCC. Roche is also evaluating vismodegib in a Phase II trial in people with operable forms of BCC.

Roche is developing vismodegib under a collaboration agreement with Curis, Inc. Vismodegib was discovered by Genentech and jointly validated by Genentech and Curis through a series of preclinical studies.

Through this collaboration, Genentech (United States), Roche (ex-United States excluding Japan) and Chugai Pharmaceuticals (Japan) are responsible for the clinical development and commercialisation of vismodegib. Curis is eligible to receive cash payments upon the successful achievement of specified clinical development and regulatory approval milestones, as well as royalties upon commercialization of vismodegib.