The US Food and Drug Administration (FDA) has approved Curis Inc's Erivedge (vismodegib) capsule for the treatment of adults with a type of skin cancer, called basal cell carcinoma (BCC), that has spread to other parts of the body or that has come back after surgery or that their healthcare provider decides cannot be treated with surgery or radiation.

Erivedge is the first and only FDA-approved medicine for people with advanced forms of the most common skin cancer. It is being developed and will be commercialised by Roche and Genentech, a member of the Roche Group, under a collaboration agreement between Curis and Genentech.

Curis earned a $10 million milestone payment from Genentech as a result of the FDA's approval of Erivedge in this indication and is also entitled to receive royalties on future sales of the product.

BCC is generally considered curable if the cancer is restricted to a small area of the skin. People with advanced BCC have tumours that have spread to other parts of the body (metastasized), or can become disfiguring and invade surrounding tissue (locally advanced). In these cases, the disease cannot be treated with surgery or radiation. Advanced BCC often results in severe deformity or impaired function of the affected organs.

“We are extremely pleased that Erivedge has been approved as a new treatment for patients suffering from advanced BCC in the US, representing the first approved treatment option for patients with this serious medical condition,” said Dan Passeri, Curis president and chief executive officer. “We continue to be pleased with Genentech and Roche's global Erivedge development efforts. In addition to the US, Roche submitted a marketing authorization application for Erivedge in the European Union and this application is currently under review by the European Medicines Agency, potentially significantly increasing patient access to Erivedge in the future.”

Erivedge is expected to be available in the United States within one-to-two weeks of approval and to be distributed through specialty pharmacies.

The FDA approval of Erivedge is based on results from ERIVANCE BCC (SHH4476g), a pivotal international, single-arm, multi-centre, two-cohort, open-label, phase II study that enrolled 104 patients with advanced BCC, including locally advanced BCC (71) and metastatic BCC (33).

The study showed Erivedge shrank lesions (objective response rate, or ORR, the primary endpoint of the study, as assessed by an independent review) in 43 percent (27/63) of patients with locally advanced BCC and 30 percent of patients (10/33) with metastatic BCC. The median duration of response was 7.6 months.

According to the American Cancer Society, BCC accounts for approximately 80 per cent of all diagnosed skin cancers. The disease is generally considered curable if the cancer is restricted to a small area of the skin. However, in a small group of people, if the disease is left untreated or recurs in the same location after surgery or radiotherapy, it may become locally advanced and invade further into surrounding areas such as sensory organs (ears, nose and eyes), bone, or other tissues.

Depending on the location of the lesion, some cases of advanced BCC can be disfiguring, and treatment with surgery or radiation can lead to the loss of sensory organs and their functions such as eyesight or hearing. In a small proportion of patients, BCC can metastasize, spreading to other parts of the body. Abnormal signalling in the Hedgehog pathway is implicated in the more than 90 per cent of BCC cases.

Erivedge is designed to selectively inhibit signalling in the Hedgehog pathway by binding to a protein called Smoothened. The Hedgehog signalling pathway plays an important role in regulating proper growth and development in the early stages of life and becomes less active in adults.

Genentech is also evaluating Erivedge in a phase II trial in patients with operable forms of BCC, which opened for patient enrollment in October 2010. Furthermore, Erivedge is being evaluated by third-party investigators in a number of other cancers and in people with BCC who have Gorlin syndrome, a condition that affects many areas of the body and increases the risk of developing BCC.

Under the ongoing collaboration agreement between Genentech, a wholly owned member of the Roche Group, and Curis, Erivedge (vismodegib, GDC-0449, RG3616) was discovered by Genentech and was jointly validated by the parties through a series of preclinical studies. Pursuant to this collaboration, Genentech and Roche are responsible for clinical development, and Genentech (US), Roche (Ex-US excluding Japan) and Chugai Pharmaceuticals (Japan) are responsible for commercialization of Erivedge. Curis is eligible to receive cash payments upon the successful achievement of specified clinical development and regulatory approval milestones, as well as royalties assuming successful commercialization of Erivedge by Genentech and its sublicensees, which include Roche and Chugai.