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

Successful Alternative Administration of Sonidegib in a Patient with Gorlin-Goltz Syndrome and Dysphagia: A Case Report. (PubMed, Case Rep Dermatol) - "Approved HH pathway inhibitors, like vismodegib and sonidegib, are supplied in capsule form. Patients who suffer from dysphagia may have problems swallowing these medications. This case report describes the successful use of an alternative administration of sonidegib in a patient with Gorlin-Goltz syndrome and dysphagia."

Journal • Basal Cell Carcinoma • Gastrointestinal Disorder • Non-melanoma Skin Cancer • Oncology • Otorhinolaryngology

Clinical Outcomes of Sonidegib in Vismodegib-Exposed Locally Advanced Basal Cell Carcinoma: Insights From a Multicenter Descriptive Study. (PubMed, J Skin Cancer) - "No novel AEs have been documented, and they were managed through dose adjustments or temporary interruptions. These findings suggest that sonidegib could still show efficacy and may still serve as a viable second-line option after prior Hh pathway suspension."

Clinical data • Journal • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

Mohs Surgery Outcomes for Basal Cell Carcinoma Treated With Neoadjuvant Sonic Hedgehog Inhibitor. (PubMed, Int J Dermatol) - No abstract available

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

Circumventing exenteration for giant basal cell carcinoma by neoadjuvant Sonidegib. (PubMed, Eye (Lond)) - No abstract available

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

Phase 1/1b study of azacitidine and hedgehog pathway inhibitor sonidegib in patients with myeloid neoplasms. (PubMed, Cancer) - "Our study shows that AZA + SON are a safe combination in a patient with MN. Similar to other hedgehog inhibitors, this combination yielded limited response rate in patients with myeloid neoplasms."

Journal • P1 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • CNS Disorders • Constipation • Cough • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Insomnia • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Oncology • Respiratory Diseases • Sleep Disorder

Regulatory Signaling Pathways in Ovarian Cancer Stem Cells: Their Role in Tumor Progression and Therapeutic Strategies. (PubMed, Iran Biomed J) - "Various therapeutic agents have been studied to target these pathways in OCSCs, including γ-secretase inhibitors (nirogacestat), smoothened inhibitors (vismodegib and sonidegib), and Wnt/β-catenin inhibitors (PRI-724 and ipafricept). To improve treatment outcomes, strategies involving combination approaches and personalized treatments need to be explored. This review aims to summarize current evidence on the role of Wnt/β-catenin, Notch, and Hedgehog signaling pathways in OCSCs and their therapeutic implications in ovarian cancer."

Journal • Oncology • Ovarian Cancer • Solid Tumor

Aberrant Activation of the Hedgehog Pathway in Cutaneous Melanoma: Therapeutic Potential of Pharmacological Inhibitors. (PubMed, Int J Mol Sci) - "Profiling inflammatory mediators revealed significant modulation upon treatment with SMO inhibitors, possibly affecting chemotactic and immune functions. Collectively, these findings provide deeper insight into the role of the Hh pathway in melanoma and support the potential repurposing of Hh inhibitors as therapeutic agents for melanoma."

Journal • Cutaneous Melanoma • Genetic Disorders • Melanoma • Oncology • Skin Cancer • Solid Tumor • GLI2 • PTCH1

Repurposing Itraconazole in Clinical Dermato-oncology Beyond Conventional Antifungal Use- A review. (PubMed, Clin Exp Dermatol) - "Unlike conventional SHH pathway inhibitors such as vismodegib and sonidegib, which exert their effect via direct binding to the transmembrane protein Smoothened (SMO), itraconazole impedes intracellular SMO trafficking, thereby attenuating downstream pathway activation. Despite encouraging preliminary evidence, further investigation through randomized controlled trials is warranted to optimize dosage regimens, define therapeutic windows, and explore synergistic potential with existing oncologic agents. The repositioning of itraconazole exemplifies the expanding interface between dermatology and oncology and highlights the promise of drug repurposing in cutaneous malignancies."

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

Alcian blue-positive stromal phenotype in basal cell carcinoma is associated with progression on first-line hedgehog inhibitors. (PubMed, J Pathol Clin Res) - "Hedgehog pathway inhibitors (HHIs; vismodegib/sonidegib) constitute standard first-line treatment, yet individual responses vary and no histopathological biomarker predicting therapeutic outcome exists. Our findings identify diffuse AB-positive stroma as a readily detectable feature of histologically aggressive BCC and as a candidate biomarker associated with progression under HHI treatment. Because AB staining is routine, inexpensive, and easily standardized, this phenotype represents an immediately implementable readout for prospective validation and a potential link between extracellular-matrix remodeling and therapy resistance in BCC."

Biomarker • Journal • Retrospective data • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology • Skin Nodular Basal Cell Carcinoma

Resistance to SMO Inhibitors in Advanced Basal Cell Carcinoma: A Case Highlighting the Role of Molecular Tumor Profiling. (PubMed, Int J Mol Sci) - "Accordingly, current targeted therapies for locally advanced, unresectable, or metastatic BCC focus on SMO inhibition, using orally administered drugs such as vismodegib and sonidegib. To our knowledge, this is the first report documenting a sonidegib resistance mechanism in BCC that is independent of HH pathway mutations. This case highlights the complexity of resistance mechanisms to HH inhibitors and underscores the critical need for comprehensive molecular tumor profiling prior to initiating targeted therapy."

Journal • Basal Cell Carcinoma • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer • FGFR1 • NOTCH1 • PTCH1 • SMO • TP53

An Updated Comprehensive Pharmacovigilance Study of Drug- Induced Breast Cancer Based on FDA Adverse Event Reporting System Data. (PubMed, Curr Med Chem) - "Targeted therapies of lapatinib, fulvestrant, and endocrine agents (letrozole or ribociclib) show disproportionately high AE reporting odds ratios, necessitating vigilant monitoring. HER2-positive BC treatments (trastuzumab) and TNBC agents (sonidegib) exhibit distinct risk profiles, advocating for personalized risk-benefit assessments for BC patients."

Adverse events • Journal • Breast Cancer • Cardiovascular • HER2 Breast Cancer • HER2 Positive Breast Cancer • Oncology • Rheumatology • Solid Tumor • Triple Negative Breast Cancer • HER-2

Vismodegib and Sonidegib Are Associated With an Elevated Reporting Odds of Squamous Cell Carcinoma; a Comparative Pharmacovigilance Study of the FDA Adverse Events Reporting System (FAERS). (PubMed, Australas J Dermatol) - "Vismodegib has a significantly higher ROR for alopecia and taste symptoms, while sonidegib was associated with neutropenia, which is a novel finding. Both HIs are associated with an increased ROR for cSCC, which persists despite stringent controls and does not parallel total cSCC cases reported to FAERS. These data suggest that HIs increase the risk of cSCC above the baseline expected in this patient population."

Adverse events • Journal • Alopecia • Basal Cell Carcinoma • Hematological Disorders • Immunology • Musculoskeletal Pain • Neutropenia • Non-melanoma Skin Cancer • Oncology • Pain • Squamous Cell Carcinoma

Real-World Treatment Patterns of Patients with Basal Cell Carcinoma Using Sonidegib and Vismodegib: Discontinuation Rates and Clinical Conditions During Treatment. (PubMed, Dermatol Ther (Heidelb)) - "In the real-world setting, sonidegib-treated patients remained on treatment longer than vismodegib-treated patients and were less likely to experience pharmacologically relevant clinical conditions."

HEOR • Journal • Real-world evidence • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

SONIB: A Study to Evaluate Neoadjuvant Sonidegib Followed by Surgery or Imiquimod in the Management of Basal Cell Carcinoma (clinicaltrials.gov) - P2 | N=16 | Completed | Sponsor: Melanoma Institute Australia | Recruiting ➔ Completed | Trial completion date: Jun 2026 ➔ Mar 2025 | Trial primary completion date: Jun 2026 ➔ Mar 2025

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

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