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• The FDA approved vismodegib (Erivedge) for the treatment of metastatic basal cell carcinoma (BCC) in adults or locally advanced basal cell carcinoma that has recurred following surgery or in those who are not candidates for surgery, and who are not candidates for radiation.

Efficacy was confirmed in a single-arm, parallel cohort trial following 104 participants in which patients were given 150 mg of vismodegib daily. Of these, 96 had confirmed BCC (33 with metastatic basal cell carcinoma [mBCC] and 63 with locally advanced basal cell carcinoma [laBCC]) and were evaluated. Median age was 62 years, 61% were male, and 97% had an ECOG performance status of 0 or 1.

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Of the participating patients, 21% had Gorlin sydrome; 66% had locally advanced disease; and 34% had metastatic disease. Among those with mBCC, 97% were previously treated; among laBCC, 94% were previously treated.

The primary endpoint was objective response rate (ORR) assessed by an independent review facility. For laBCC, tumor response criteria included assessment of tumor size, the presence or absence of ulceration, and biopsy of local disease sites. For mBCC, complete response criteria included tumor biopsies demonstrating no pathologic evidence of BCC.

The ORRs were 30.3% (95% confidence interval [CI]: 15.6, 48.2) and 42.9% (95% CI: 30.5, 56.0) in patients with mBCC and laBCC, respectively. All responses in the mBCC cohort were partial responses, and of 63 evaluable patients in the laBCC arm, 13 (20.6%) had complete responses and 14 (22.2%) had partial responses. Median response duration was 7.6 months (96% CI: 5.6, not estimable) and 7.6 months (95% CI: 5.6, 9.7) for patients with mBCC and laBCC, respectively.

Healthcare professionals should monitor for pregnancy status before administering vismodegib, educate patients about potential risks to the embryo/fetus, and advise nonpregnant patients to use highly effective contraception during therapy with vismodegib.

Vismodegib works by inhibiting the Hedgehog pathway, an important embryonic developmental pathway. Previous studies in rats have shown that vismodegib exposure during organogensis results in embryo-fetal death at higher exposures and severe birth defects at exposures within the range of the recommended human dose. See the video above for more information about the Hedgehog pathway.

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