The US Food and Drug Administration (FDA) has approved sonidegib (Novartis’s Odomzo) as a treatment for patients with locally advanced basal cell carcinoma that has recurred following surgery or radiation therapy, as well as for patients who are not candidates for surgery or radiation therapy.
Sonidegib is taken once a day and works by inhibiting a molecular pathway known as the Hedgehog pathway, which is active in basal cell cancers. By suppressing the pathway, the drug may reduce the growth of cancerous lesions. It is the second drug that interferes with this pathway to be approved for basal cell carcinoma by the FDA in recent years; vismodegib (Erivedge) was approved in 2012.
In a clinical trial, the tumours of 58% of patients taking sonidegib 200mg daily shrank or disappeared, with the effects lasting from 1.9 months to 18.6 months.
Sonidegib will carry a warning that it may cause death or severe birth defects in a developing foetus when administered to a pregnant woman. The drug also has the potential to cause serious musculoskeletal-related side effects, including increased serum creatine kinase levels (with rare reports of rhabdomyolysis), muscle spasms and myalgia.
Common side effects with sonidegib include muscle spasms, alopecia, dysgeusia (distortion in the sense of taste), fatigue, nausea, musculoskeletal pain, diarrhoea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting and pruritus.
The European Medicines Agency issued a positive opinion on sonidegib on 25 June 2015.