FDA approves Odomzo (sonidegib, formerly LDE225) to treat basal cell carcinoma.- Novartis

Novartis announced the FDA has approved Odomzo (sonidegib, formerly LDE225) 200 mg capsules 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 Odomzo approval was based on the demonstration of a durable objective response rate (ORR) in an international, multi-center, double-blind, randomized, two-arm, non-comparative trial in patients with laBCC not amenable to local therapy or metastatic basal cell carcinoma (mBCC).

Patients with laBCC treated with Odomzo 200 mg (n=66) were followed for at least 12 months unless discontinued earlier. The ORR was 58% (95% confidence interval: 45, 70), consisting of 5% (n=3) complete responses (CR) and 53% (n=35) partial responses (PR). A pre-specified sensitivity analysis using an alternative definition for CR, defined as at least a PR according to MRI and/or photography and no evidence of tumor on biopsy of residual lesion, yielded a CR rate of 20%. Among the 38 patients with an objective response, 31 patients (82%) have ongoing responses ranging from at least 1.9 to 18.6 months and the median duration of response has not been reached. The most serious risks of Odomzo are embryofetal toxicity and musculoskeletal adverse reactions including rhabdomyolysis.

Comment: Odomzo was CHMP recommended in June 2015.

Comment: Odomzo will compete with hedgehog inhibitor Erivedge (vismodegib, licensed from Curis) from Roche. This became the first FDA-approved therapy for the treatment of locally advanced or metastatic basal cell carcinoma early in 2012, which was followed by an EU conditional approval in 2013, and until now it enjoyed no competition in this niche market.