An oral agent developed by Novartis Pharmaceuticals Corporation has been approved for the treatment of refractory patients with locally advanced basal cell carcinoma (laBCC). Patients who have undergone unsuccessful surgery or radiation treatment, or those who are not eligible for either, can initiate treatment with the sonic hedgehog inhibitor sonidegib (Odomzo). The FDA approval followed results of a randomized, double-blind, multi-center, 2-arm study in patients with laBCC or metastatic BCC. The approval was contingent on a durable objective response rate (ORR).
The phase 2 BOLT trial enrolled 230 patients randomized to receive 800 mg or 200 mg of sonidegib until progression or unacceptable toxicity was observed. Eighty-four percent of trial enrollees had locally advanced disease. Most patients (76%) had received prior therapy for treatment of BCC, and approximately half of these patients (56%) had aggressive histology. The trial results showed a durable ORR of 58% (95% CI, 0.45—0.70) in 66 patients randomized to receive the 200 mg dose of sonidegib, with 3 complete responses and 35 partial responses. While the disease progressed in 7 of the 38 responders, response in 4 of the 7 patients lasted at least 6 months. Patients (128) on the 800-mg arm had an ORR of 44% (95% CI, 0.35–0.53).
The most serious risks of sonidegib are rhabdomyolysis and embryofetal toxicity.
Reference
Sonidegib. FDA website. http://www.fda.gov/drugs/informationondrugs/approveddrugs/ucm455865.htm. Accessed July 27, 2015.
Real-World Study Reveals Key Insights into DLBCL Treatment Patterns, Outcomes
April 18th 2024A recent study offers valuable insights into the characteristics, treatment patterns, and outcomes of diffuse large B-cell lymphoma (DLBCL) in patients across different lines of therapy, providing a look into the landscape of DLBCL management.
Read More
Pegcetacoplan for PNH More Cost-Effective Than Anti-C5 Monoclonal Antibodies
April 18th 2024A cost-utility analysis conducted from the perspective of the Italian health system found that pegcetacoplan was more effective and less costly than 2 complement 5 (C5) inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Read More
Oncology Onward: A Conversation With Penn Medicine's Dr Justin Bekelman
December 19th 2023Justin Bekelman, MD, director of the Penn Center for Cancer Care Innovation, sat with our hosts Emeline Aviki, MD, MBA, and Stephen Schleicher, MD, MBA, for our final episode of 2023 to discuss the importance of collaboration between academic medicine and community oncology and testing innovative cancer care delivery in these settings.
Listen
Many Patients With Psoriasis in Clinical Trials Experience Nocebo Effects, Study Finds
April 18th 2024Half of patients exposed to placebo in clinical trials experienced adverse events (AEs), which may be partially explainable by nocebo effects, according to a recent review and meta-analysis.
Read More
Low-Volume Hospitals Had Higher Reoperation Rate, Postoperative Complications in CRC
April 18th 2024Patients opting for elective colorectal surgery to address colorectal cancer (CRC) could have different rates of reoperation and postoperative complications based on the size of the hospital.
Read More