MedWire News: The US Food and Drug Administration (FDA) has approved vismodegib for treatment of metastatic basal cell carcinoma (BCC).
Vismodegib is the first drug to be approved by the FDA for treatment of patients with advanced or metastatic BCC and is also the first-in-class Hedgehog pathway inhibitor. Malfunctions in the Hedgehog pathway are implicated in many cancers including BCC.
"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 (FDA Center for Drug Evaluation and Research, Beltsville, Maryland, USA) in a press statement.
"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," he added.
The drug was reviewed under the FDA's priority review program, which allows drugs that have the potential to dramatically improve treatment to be given priority over other drugs and be reviewed within 6 months. Vismodegib has been approved in advance of the prescription user fee goal date of March 2012.
The approval is based on the results of a multi-center trial involving 96 patients with advanced or metastatic BCC who were not candidates for surgery or radiotherapy. In total, 30% of metastatic and 43% of locally advanced BCC patients achieved complete/partial shrinkage or disappearance of the cancerous lesions after treatment with vismodegib.
Side effects were monitored and were most commonly muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, and taste distortion.
The drug is also being released with a boxed warning to alert both clinicians and patients to the high risks for fetal death or birth deformity associated with taking the drug in pregnancy.
Vismodegib is not currently approved outside the USA, but a phase II safety study is currently enrolling patients in the EU and other countries. It is also being evaluated in the USA as a potential treatment for people with less severe forms of BCC.
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US FDA website
