The US Food and Drug Administration has approved Novartis’ once-daily pill Odomzo for locally advanced basal cell carcinoma that has returned following surgery or radiation therapy, or for patients unable to have these interventions.

BCC consists of abnormal, uncontrolled growths or lesions that arise in the skin's basal cells, which line the deepest layer of the epidermis, and accounts for more than 80% of non-melanoma skin cancers.

Odomzo (sonidegib) is an oral, selective smoothened inhibitor that regulates the hedgehog signalling pathway, known to play a critical role in stem cell maintenance and tissue repair as well as in advanced BCC.

Approval was based on clinical trials showing an overall response rate - the percentage of patients who experienced partial shrinkage or complete disappearance of their tumour - of 58% in those given the drug. This effect lasted at least 1.9 to 18.6 months, and around half of the responding patients’ tumour shrinkage lasted six months or longer, the FDA noted.

Boxed warning

On the downside, Odomzo carries a Boxed Warning that the drug may cause death or severe birth defects in a developing fetus when administered to a pregnant woman, and the incidence of musculoskeletal adverse reactions in patients taking part in trials was 68%, with 9% reported as grade 3 or 4. 

Also, adverse reactions occurring in more than 10% of patients treated with Odomzo were muscle spasms, alopecia, dysgeusia, fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting, and pruritus.

According to the National Cancer Institute, the number of new cases of non-melanoma skin cancer appears to be increasing every year. Odomzo is now the second hedgehog pathway inhibitor approved by the FDA for BCC in recent years, following the nod for Roche/Curis’ Erivedge (vismodegib) back in 2012.