The European Commission has granted conditional approval to Erivedge ( Vismodegib ) for the treatment of adult patients with symptomatic metastatic basal cell carcinoma ( BCC ) or locally advanced BCC inappropriate for surgery or radiotherapy.
This approval makes Erivedge, a capsule taken once-a-day, the first licensed medicine for patients in the European Union with this disfiguring and potentially life-threatening form of skin cancer.
A conditional marketing authorisation is granted to medicinal products with a positive benefit/risk assessment that satisfy an unmet medical need and whose availability would result in a significant public health benefit.
Under the provisions of the conditional approval, Roche will provide additional data on Erivedge in advanced BCC from a global safety study.
Basal cell carcinoma is generally considered curable when confined to the skin. However, in some cases the disease will invade surrounding tissue ( locally advanced ) or spread to other parts of the body ( metastatic BCC ) in a manner that cannot be effectively treated with surgery or radiation.
In January 2012, Erivedge became the first licensed medicine for patients with advanced basal cell carcinoma in the United States when the FDA ( Food and Drug Administration ) approved it under the priority review programme that provides for an expedited review of drugs that offer major advances in treatment.
This conditional approval is based on findings from the primary analysis ( 26 November 2010 ) in the pivotal ERIVANCE BCC phase II single-arm study which enrolled 104 patients with advanced BCC ( 71 had locally advanced and 33 had metastatic disease ) from 31 study sites in the U.S., Australia and Europe.
The study showed Erivedge substantially shrank lesions ( objective response rate, or ORR ) in 43% ( 27/63 ) of patients with locally advanced basal cell carcinoma and 30% of patients ( 10/33 ) with metastatic basal cell carcinoma, as assessed by independent review. The median duration of response was 7.6 months.
The most common adverse events included muscle spasms, hair loss, altered taste sensation, fatigue and weight loss. Serious adverse events ( SAEs ) were observed in 26 patients ( 25% ), however of these only four ( 4% ) patients had SAEs that were considered to be related to treatment with Erivedge.
The safety profile of Erivedge is being further assessed in STEVIE, a global, single-arm, open-label multicentre study of patients with advanced forms of basal cell carcinoma. The study aims to enrol 1,200 patients. An interim analysis from STEVIE presented at ASCO 2013 confirmed a similar safety profile to that observed in the ERIVANCE BCC study.
Basal cell carcinoma is the most common type of skin cancer in Europe, the United States and Australia. It is a slow-growing skin cancer typically caused by long term and/or occasionally excessive exposure to the sun and accounts for as many as 80% of all non-melanoma skin cancers.
In advanced basal cell carcinoma, if the disease is left untreated or recurs in the same location after surgery or radiotherapy, it may advance 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 basal cell carcinoma can be disfiguring, and treatment with surgery or radiotherapy can lead to the loss of sensory organs and their functions such as eyesight or hearing.
Vismodegib is a Hedgehog pathway inhibitor, a cancer medicine designed to selectively target and block abnormal activity in a cell growth pathway, known as the Hedgehog signaling pathway, which helps to regulate growth and development in the early stages of life.
The Hedgehog pathway usually becomes less active as a person matures. However, mutations may reactivate the pathway, causing it to work incorrectly. These mutations are thought to occur in nearly all ( at least 90% ) cases of basal cell carcinoma. ( Xagena )
Source: Roche, 2013