Skin cancer drug Odomzo gets FDA approval

July 27, 2015

FDA has approved sonidegib (Odomzo, Novartis) for locally advanced basal cell carcinoma.

FDA has approved sonidegib (Odomzo, Novartis) for locally advanced basal cell carcinoma.

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Basal cell carcinoma (BCC) is a type of skin cancer characterized by abnormal, uncontrolled growths or lesions that arise in the skin’s basal cells. BCC accounts for approximately 80% of non-melanoma skin cancers. When BCC spreads from where it originally started, it is called locally advanced and can be highly disfiguring. Although BCC rarely becomes advanced, there are only a few treatment options available for this stage of the disease.

Odomzo is a once daily, oral, selective smoothened (SMO) inhibitor that works by inhibiting a molecular pathway, called the Hedgehog pathway, which is active in basal cell cancers. The drug acts to suppress the pathway in order to stop or reduce the growth of cancerous lesions. Odomzo 200-mg capsules are FDA approved for the treatment of adult patients with locally advanced basal cell carcinoma (laBCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.

"The FDA approval of Odomzo offers a new and non-invasive treatment option for a potentially devastating disease that is hard to treat and can be disfiguring," said Bruno Strigini, President, Novartis Oncology. "Odomzo is an important addition to our growing portfolio of targeted treatments for advanced skin cancers and underscores our commitment to developing and bringing to market new options for patients."

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The efficacy of Odomzo was established in a multicenter, double-blind clinical trial, in which participants with locally advanced BCC were randomly assigned to receive with 200 mg daily or 800 mg daily of Odomzo. Results demonstrated that 58% of patients treated with Odomzo 200 mg had their tumors shrink or disappear, which approximately half of the responder’s tumor shrinkage lasting 6 months of longer. The 800-mg dose showed similar response rates but side effects were more common at this dose.

Muscle spasms, hair loss, distorted sense of taste, fatigue, musculoskeletal pain, diarrhea, decreased weight, and decreased appetite, were the most common side effects associated with the use of the 200-mg dose of Odomzo. Odomzo also carries a Boxed Warning alerting healthcare professionals that Odomzo may cause death or severe birth defects in a developing fetus when administered to a pregnant woman.

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