Odomzo (sonidegib) / Sun Pharma, Novartis - LARVOL DELTA

U0126 induces osteoclast differentiation via the p38-NFATc-1 signaling pathway. (PubMed, Tissue Cell) - "Furthermore, U0126 can rescue delayed tooth eruption caused by LDE225 (a Shh inhibitor) suppression of the p38 signaling pathway...Our study may provide a scientific foundation for dental treatment strategies to enhance osteoclast function in addressing tooth eruption issues, such as impacted teeth, delayed eruption of permanent teeth, and cranioclavicular dysplasia syndrome (CCD). It will also serve as a method for future research into novel approaches to treating disorders linked to bone metabolism."

Journal • Metabolic Disorders • Osteoporosis • Rheumatology • NFATC1

Postauthorization safety study (NISSO) assessing the long-term safety profile of sonidegib in patients with locally advanced basal cell carcinoma: interim analysis from the Spanish cohort. (PubMed, Actas Dermosifiliogr) - P | "Most patients experienced grade ≤ 2 common TEAEs, such as muscle spasms, dysgeusia, and alopecia that mostly manifested 3 months into therapy. These interim results confirm the safety profile observed in the BOLT study, demonstrating that sonidegib is well-tolerated in a real-world setting in Spain."

Journal • Alopecia • Basal Cell Carcinoma • Immunology • Non-melanoma Skin Cancer • Oncology

Treatment of basosquamous carcinoma with sonidegib in a patient with HIV. (PubMed, Actas Dermosifiliogr) - No abstract available

Journal • Human Immunodeficiency Virus • Infectious Disease • Oncology

Adverse events in patients aged ≥ 85 vs < 85 years with advanced basal cell carcinoma treated with sonidegib. (PubMed, Actas Dermosifiliogr) - No abstract available

Adverse events • Journal • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

Molecular insights into response and resistance in children with relapsed/refractory brain tumors on ribociclib combination therapy: results from SJDAWN (SNO 2025) - P1 | "Safety, tolerability, and recommended phase 2 dosing for ribociclib combinations with gemcitabine (A), trametinib (B), and sonidegib (C) were previously reported. Collectively, ribociclib combination therapy exhibited clinical benefit in select patients before resistance emerged. cfDNA assessment enabled early detection of resistance, unveiling a window for therapeutic adjustment."

Clinical • Combination therapy • Brain Cancer • Ependymoma • Glioma • High Grade Glioma • Medulloblastoma • Oncology • Solid Tumor • CCND2 • MYCN • TP53

A phase II trial combining pulsed sonidegib (soni) and cemiplimab (cemi) in advanced basal cell carcinoma (aBCC) (ESMO 2025) - P2 | "Conclusions Short term pulsed Soni and Cemi is feasible, shows promising clinical efficacy associated with improved antigen presentation accompanied by an IFNγ response. Unexpectedly, Soni alone already resulted in systemic immune activation."

Metastases • P2 data • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology • CD4 • CD8 • IFNG • IL12A • IL18 • TNFA

A Sonidegib Experience in the Treatment of Basal Cell Carcinoma with Systemic Lupus Erythematosus: A Case Report. (PubMed, Clin Cosmet Investig Dermatol) - "Presently, there's a lack of documented clinical use of sonidegib in BCC patients alongside LE, particularly due to its unclear immunomodulatory impact on existing autoimmune diseases. In this report, we present a case involving a BCC patient and systemic lupus erythematosus (SLE), who received sonidegib treatment yielding positive results without simultaneously triggering lupus erythematosus activity."

Journal • Basal Cell Carcinoma • Discoid Lupus Erythematosus • Genetic Disorders • Immunology • Inflammatory Arthritis • Lupus • Non-melanoma Skin Cancer • Oncology • Skin Cancer • Systemic Lupus Erythematosus

SONIB: A Study to Evaluate Neoadjuvant Sonidegib Followed by Surgery or Imiquimod in the Management of Basal Cell Carcinoma (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Melanoma Institute Australia | Trial completion date: Jun 2025 ➔ Jun 2026 | Trial primary completion date: Jun 2025 ➔ Jun 2026

Trial completion date • Trial primary completion date • Basal Cell Carcinoma • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer

Molecular insights into response and resistance in children with relapsed/refractory brain tumors on ribociclib combination therapy: results from SJDAWN (WFNOS 2025) - P1 | "Safety, tolerability, and recommended phase 2 dosing for ribociclib combinations with gemcitabine (A), trametinib (B), and sonidegib (C) were previously reported. Collectively, ribociclib combination therapy exhibited clinical benefit in select patients before resistance emerged. cfDNA assessment enabled early detection of resistance, unveiling a window for therapeutic adjustment."

Clinical • Combination therapy • Brain Cancer • Ependymoma • Glioma • High Grade Glioma • Medulloblastoma • Oncology • Solid Tumor • CCND2 • MYCN • TP53

Real-world safety profile of sonidegib: a disproportionality analysis based on the FDA adverse event reporting system. (PubMed, Front Oncol) - "This study analyzed the real-world safety of sonidegib and emphasized the importance of continuous monitoring during the early stages of treatment. These findings provide important safety information for clinicians, but further research is needed to validate these results."

Adverse events • Journal • Real-world evidence • Basal Cell Carcinoma • Infectious Disease • Musculoskeletal Pain • Nephrology • Non-melanoma Skin Cancer • Oncology • Pain • Pneumonia • Respiratory Diseases • Septic Shock

A review of the safety, efficacy, and administration of hedgehog inhibitors for the treatment of advanced basal cell carcinoma: an expert consensus panel. (PubMed, Dermatol Online J) - "Sonidegib and vismodegib have similar efficacy in treating advanced BCC, but sonidegib has lower rates and a greater delay in onset of AEs. Sonidegib has a significantly greater volume of distribution and half-life than those of vismodegib. Dosing interruptions have not been shown to reduce the efficacy of HHIs, and L-carnitine supplementation can help reduce the incidence of muscle spasms."

Journal • Review • Basal Cell Carcinoma • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer

Disproportionality analysis comparing safety profiles of sonidegib and vismodegib based on the FAERS database (Nature) - "This study provides an overview of HPIs-associated suspected AEs based on real-world data from the FAERS database and reveals divergent reporting patterns between sonidegib and vismodegib. We identified 25 SOCs affected by AEs associated with HPIs, predominantly in general disorders and administration site, musculoskeletal and connective tissue, gastrointestinal and nervous system. The analysis results were consistent with findings of previous observation and clinical trials. Additionally, we discovered unexpected significant AEs, such as second primary malignancies, disorders of special sensory organs, complete atrioventricular block, and so on. HPIs are a novel class of anticancer drugs, and more research is needed to characterize their safety profile."

Adverse events • Basal Cell Carcinoma • Medulloblastoma • Melanoma • Small Cell Lung Cancer

PersoMed-I: Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma (clinicaltrials.gov) - P2 | N=20 | Terminated | Sponsor: European Organisation for Research and Treatment of Cancer - EORTC | Trial completion date: Dec 2025 ➔ Sep 2025 | Active, not recruiting ➔ Terminated | Trial primary completion date: Dec 2025 ➔ Jun 2025; Poor accrual

Trial completion date • Trial primary completion date • Trial termination • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor • BRCA2 • MYCN • TP53

A case of basal cell nevus syndrome with a SUFU mutation. (PubMed, Dermatol Online J) - "Rarely, this condition is related to a suppressor of fused gene mutation, which occurs downstream from Smoothened, and is unresponsive to Smoothened inhibitors including vismodegib and sonidegib. Genetic testing was negative for patched 1 and patched 2 mutations but positive for a heterozygous suppressor of fused mutation. Patients with basal cell nevus syndrome should be treated with surgical excision, counseled on sun protection, screened and monitored for complications, and treated with vismodegib (if associated with patched 1 mutation) or itraconazole (if associated with suppressor of fused mutation)."

Journal • Basal Cell Carcinoma • Brain Cancer • Dermatopathology • Medulloblastoma • Oncology • Rare Diseases • Solid Tumor • PTCH1 • PTCH2 • SUFU

Evaluation of Sonidegib Cytoreductive Activity in Locally Advanced Basal Cell Carcinoma Treatment: A Delphi Consensus. (PubMed, Int J Dermatol) - "In the absence of solid scientific evidence, this article offers consensus-based recommendations on which patients may benefit from cytoreductive HHI, how to conduct the cytoreductive therapy, how to assess response to it and how to perform the surgery."

Journal • Basal Cell Carcinoma • Dermatology • Non-melanoma Skin Cancer • Oncology

Multicenter Retrospective Case Series on the Real-World Experience With Sonidegib for the Management of Gorlin Syndrome. (PubMed, Australas J Dermatol) - "Our data on the use of sonidegib for GS in clinical practice show similar response rates to those reported in previous studies and improved tolerability compared to pivotal studies. Regimen modifications are frequently used in clinical practice to improve AEs. Although the alternate-day dose is the most common modification, dermatologists also resort to off-label regimens for this purpose."

Journal • Real-world evidence • Retrospective data • Alopecia • Basal Cell Carcinoma • Dermatology • Immunology • Non-melanoma Skin Cancer • Oncology

Disproportionality analysis comparing safety profiles of sonidegib and vismodegib based on the FAERS database. (PubMed, Sci Rep) - "However, as a spontaneous reporting system, FAERS cannot establish causal relationships. These findings should therefore be considered hypothesis-generating and warrant further research."

Clinical • Journal • Alopecia • Basal Cell Carcinoma • Breast Cancer • Eye Cancer • Immunology • Non-melanoma Skin Cancer • Oncology • Otorhinolaryngology • Solid Tumor • Squamous Cell Carcinoma • Triple Negative Breast Cancer

Ring expansion of bicyclo[1.1.0]butyl ketones to bicyclo[2.1.1]hexenes using ketone as both activating and reacting groups. (PubMed, Nat Commun) - "A series of transformations have been carried out on bicyclo[2.1.1]hexenes to synthesize bicyclo[2.1.1]hexane/hexene analogues of sonidegib, and the physicochemical properties and anti-tumor activities of these analogues have been investigated. In addition, this approach enables the discovery of aggregation-induced emission properties in this distinctive ring skeleton."

Journal • Oncology

Gastrointestinal adverse events associated with Hedgehog pathway inhibitors: a disproportionality analysis. (PubMed, Int J Pharm Pract) - "HPIs had triggered various types of GIAEs. Liver injuries were a safety concern for the HPI-treated patient, which was not listed by the USFDA. Further researches are needed to confirm these findings."

Adverse events • Journal • Basal Cell Carcinoma • Liver Failure • Non-melanoma Skin Cancer • Oncology

Co-priming sonic hedgehog inhibitor sonidegib with plerixafor induces tumor microenvironment transformation that enhances fluorescent nanoparticle deposition in pancreatic tumor xenograft Free (AACRPanCa 2025) - "Nanomedicines have seen widespread use in several malignancies including pancreatic cancer where standard-of-care chemotherapy regimens regularly employ nanomedicines including nab-paclitaxel and liposomal irinotecan. This combination may be explored further to probe anti-tumor efficacy when combined with nanoparticle-associated chemotherapy currently employed for treating pancreatic cancer. Future investigations will evaluate the pro-immune effects of plerixafor in this co-priming strategy to improve outcomes with contemporary immune checkpoint therapeutics."

Biomarker • Tumor microenvironment • Fibrosis • Immunology • Oncology • Pancreatic Cancer • Solid Tumor

Vismodegib treatment in locally advanced basal cell carcinoma limited to the facial region: a single-center experience. (PubMed, BMC Cancer) - "Vismodegib demonstrated high efficacy in treating patients with laBCC, with a significant proportion achieving CR. However, long-term treatment was associated with an increased incidence of AEs, highlighting the need for careful monitoring of safety in prolonged therapies."

Journal • Basal Cell Carcinoma • Genetic Disorders • Non-melanoma Skin Cancer • Skin Cancer • Solid Tumor

Safety and Efficacy of Vismodegib and Sonidegib in Advanced Basal Cell Carcinoma of the Head and Neck: A Systematic Review and Meta-Analysis. (PubMed, Head Neck) - "This systematic review and meta-analysis demonstrated a high efficacy of vismodegib and sonidegib for treating advanced BCCs in the head and neck region, suggesting a viable option when surgery is not feasible. Further high-quality research is necessary to optimize treatment outcomes for this patient population."

Journal • Retrospective data • Basal Cell Carcinoma • Fatigue • Head and Neck Cancer • Non-melanoma Skin Cancer • Oncology • Solid Tumor

Non-canonical Hedgehog signalling regulates CD8 microenvironment (CICON 2025) - "Using murine models of colorectal cancer treated with the clinically approved Smo inhibitor Sonidegib, and bespoke knockout mouse models of central Hh pathway components as well as tumour biopsies from patients treated with Hh inhibitors in the clinic, we show that Smo inhibition significantly impairs CD8+ T cell infiltration into the TME...In conclusion, our study uncovers a novel mechanism controlling T cell migration in vitro and in vivo that may reveal unique therapeutic entry points for enhancing migration in adoptive cell therapies. The findings suggest that systemic Hh inhibition may inadvertently impair anti-tumour immunity by limiting CTL access to tumours, warranting a reassessment of Hh-targeting approaches in the clinic."

Basal Cell Carcinoma • Brain Cancer • Colorectal Cancer • Gastrointestinal Cancer • Medulloblastoma • Non-melanoma Skin Cancer • Solid Tumor • CD8 • RHOA

Sonidegib in Adult Patients With Locally Advanced Basal Cell Carcinoma (laBCC) in China (clinicaltrials.gov) - P4 | N=160 | Completed | Sponsor: Jemincare | Active, not recruiting ➔ Completed

Trial completion • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

Systemic SMO-Hedgehog inhibition with Sonidegib: real-life experience of the Dermatology Unit of University of Pisa (EADV 2025) - "If not feasible, alternative methods encompass radiotherapy, cryotherapy, topical agents (such as imiquimod cream), and systemic therapy. SMO-Hedgehog-inhibitor drugs, sonidegib and vismodegib, are used for difficult-to-treat basal cell carcinomas (locally advanced, multiple, or metastatic variants)... Sonidegib is an oral SMO-Hedgehog inhibitor approved for the treatment of locally advanced basal cell carcinomas (BCCs). Based on our expertise, it is a secure and effectively administered medication that provides patients with a comprehensive response or the opportunity to opt for a less invasive surgical intervention. Its application in patients with multiple BCCs may minimize surgical interventions, hence decreasing expenses and enhancing quality of life."

Clinical • Basal Cell Carcinoma • Dermatology • Non-melanoma Skin Cancer • Otorhinolaryngology • Skin Cancer