Basal cell carcinomas are caused by exposure to the sun or other forms of ultraviolet radiation.
Taken in the form of a daily pill, Odomzo (generic name sonidegib) is marketed by Novartis AG - a Swiss pharma giant with US headquarters in East Hanover, NJ.
The US Food and Drug Administration (FDA) say a trial showed the drug shrank or wiped out tumors in 58% of patients treated.
The regulators note that the drug is intended for patients with locally advanced basal cell carcinomas who are not candidates for surgery or radiotherapy, or whose tumors have recurred following such treatment.
Nonmelanoma skin cancers usually respond to treatment and rarely spread to other parts of the body.
The National Cancer Institute suggest the number of Americans diagnosed with nonmelanoma skin cancers is increasing every year.
BCCs arise in basal cells in the top layer of skin - the epidermis - usually in places that receive regular sun exposure or other forms of ultraviolet (UV) radiation. They occur most frequently on the head and neck, with the nose being the most common site.
Drug suppresses the Hedgehog molecular pathway
Locally advanced basal cell carcinomas (laBCCs) are basal cell cancers that have spread to nearby tissue (but not to the rest of the body) and do not respond to locally targeted treatment - that is, surgery and radiotherapy. They can be highly disfiguring.
Odomzo is a pill taken once a day. It works by suppressing the Hedgehog molecular pathway, which is active in basal cell cancers.
Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research at the FDA, says:
"Thanks to a better understanding of the Hedgehog pathway, the FDA has now approved two drugs for the treatment of basal cell carcinoma just in the last 3 years."
In 2012, the FDA approved Erivedge (vismodegib) for the treatment of locally advanced and metastatic basal cell carcinoma. The drug is marketed by Genentech of San Francisco, CA.
58% of patients saw their tumors shrink or disappear
Odomzo was tested in a double-blind clinical trial of patients with laBCC. The trial randomly assigned 66 patients to take 200 mg of the drug a day and 128 patients to take 800 mg a day.
The results showed that 58% of patients who took 200 mg of Odomzo a day saw their tumors shrink or disappear. This effect lasted for between 1.9 and 18.6 months, with around half of the responding patients experiencing it for at least 6 months.
There was a similar response rate in patients who took the higher dose, but the side effects were more common at this dose.
Side effects include muscle spasm, hair loss, distortion in sense of taste, nausea, fatigue, various forms of pain (headache, myalgia, musculoskeletal and abdominal pain), diarrhea, loss of weight, loss of appetite, vomiting and itching.
The drug can also cause serious musculoskeletal side effects; the FDA also mention rare reports of muscle tissue breakdown.
The Boxed Warning alerts doctors that Odomzo may cause death or severe birth defects in a developing fetus when taken by pregnant women.
Doctors should check whether female patients are pregnant before giving them the drug, warn both male and female patients about these risks and advise them to use effective contraception, say the FDA.
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