On Jan. 30, vismodegib became the first drug approved by the FDA for the treatment of advanced basal cell carcinoma.
In a pivotal phase II clinical trial, 43% of patients with locally advanced disease showed complete or partial tumor shrinkage while 30% of patients with metastatic disease demonstrated partial tumor shrinkage.1 These marked benefits led the drug to be approved more than 1 month ahead of schedule under the FDA's priority review track.
BCC is the most common skin cancer in the United States, with more than 1 million new diagnoses each year; up to 15% of those cases involve the eyelids.2 Hence, this is meaningful news for us all-as physicians, as ophthalmologists. However, this is particularly meaningful to me because of one patient I met 2 years ago, Mr. KK.
Additionally, the drug can have deleterious effects on developing fetuses, so all females should have pregnancy status verified pre-treatment, and contraceptive counseling is recommended for both female and male patients.1
Time will tell how vismodegib will perform long term. There are concerns about developing resistance to the drug after a certain period of time. And by the way, scientific advancement does not come cheap. Although unconfirmed, vismodegib is rumored to carry a pricetag of $75,000/10-month course.4
Still, I view the arrival of vismodegib (which will be commercially available in upcoming weeks) with excitement and optimism. We hope our growing understanding of molecular signaling in cancer will lead to the development of more drugs tailored to specific disease targets.
So, welcome, vismodegib. Your debut signifies hope for thousands of patients with BCC, and promise that similar drugs are on the horizon. It may be a small step in the fight against cancer, but it's a step in the right direction. I am sure that Mr. KK would agree.
By Christina Weng, MD, MBA Dr. Weng is a third-year ophthalmology resident at Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore. She will be pursuing a 2-year fellowship in vitreoretinal surgery at Bascom Palmer Eye Institute, University of Miami Miller School of Medicine. She has no financial conflicts of interest with vismodegib (Erivedge) or Genentech.
2. Char D. Tumors of the eye and ocular adnexa. American Cancer Society. 2001;1.
3. Epstein EH. Basal cell carcinomas: attack of the hedgehog. Nature Reviews Cancer. 2008;8:743-754.
4. Pollack A. FDA approves drug for an advanced skin cancer. The New York Times. Jan. 30, 2012.