The US Food and Drug Administration (FDA) has approved vismodegib (Erivedge™) for the treatment of adults with locally advanced basal cell carcinoma (BCC) who are not eligible for surgery, or radiation, or for patients with metastatic disease, under its priority review programme.
Vismodegib is available as an oral capsule that is taken once a day. It inhibits the Hedgehog pathway, a pathway that is active in most BCCs and only a few normal tissues, such as hair follicles.
The FDA approval is based on results from ERIVANCE BCC, an international, single-arm, multicentre, two-cohort, open-label, Phase II study that enrolled 104 patients with advanced BCC, including locally advanced BCC (n= 71) and metastatic BCC (n= 33).
Patients received 150 mg of vismodegib orally, once daily until disease progression or unacceptable toxicity.
The primary endpoint was objective response rate (ORR) or the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment, and was observed in 43% (27/63) of patients with locally advanced BCC and 30% of patients (10/33) with metastatic BCC, as assessed by independent review. The median duration of response was 7.6 months. [No further absolute data were available within the abstract.]
The most common adverse effects reported were muscle spasms, alopecia, weight loss, nausea, diarrhoea, fatigue, distorted sense of taste, decreased appetite, constipation, vomiting, and loss of taste function in the tongue.
In the US, vismodegib is being approved with a boxed warning alerting patients and health care professionals of the potential risk of death or severe birth effects to a foetus. Pregnancy status must be verified prior to the start of treatment and male and female patients should be warned about these risks and the need for birth control.
A marketing authorisation application (MAA) has also been submitted in the European Union (EU). A Phase II safety study is currently underway in the EU and other countries for advanced BCC.