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

A Review of Hedgehog Pathway Inhibitors in Combination Therapy for Basal Cell Carcinoma (AAD 2026) - "A total of nine studies were included, involving either vismodegib or sonidegib combined with concomitant therapy of either itraconazole, radiation therapy, photodynamic therapy (PDT), pembrolizumab, intratumoral immunotherapy ASN-002, or surgical debulking. Lastly, 75% of combination therapy studies reported a more tolerable AE when compared to monotherapy. Combination strategies represent a promising avenue to optimize efficacy, improve tolerability, and expand therapeutic options for patients with BCC."

Combination therapy • Review • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

Non-Surgical Management Options for Basal Cell and Squamous Cell Carcinomas: A Comparative Literature Review (AAD 2026) - "Topical agents (5-fluorouracil, imiquimod) were effective for superficial BCC and SCC in situ, with imiquimod achieving higher long-term tumor-free survival and 5-FU favored for field therapy...Hedgehog inhibitors (vismodegib, sonidegib) yielded responses in advanced BCC, though discontinuation was common due to class toxicities...In advanced disease, hedgehog inhibitors for BCC and cemiplimab for cSCC provide durable responses. A risk-stratified approach integrating tumor biology, site, patient values, and resources can guide individualized nonsurgical management."

Review • Basal Cell Carcinoma • Bowens Disease • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Skin Cancer

Characterizing the Spectrum of Adverse Events of Sonidegib for Basal Cell Carcinoma: A Real-World Pharmacovigilance Analysis Using the FAERS Database (AAD 2026) - "Our findings are consistent with previous reports of common AEs but also reveal a broader spectrum of real-world adverse events, including serious but less common complications. This study provides a valuable real-world perspective on the safety profile of sonidegib, complementing controlled clinical trial data and highlighting the importance of continued post-marketing surveillance."

Adverse events • Clinical • Real-world • Real-world evidence • Alopecia • Basal Cell Carcinoma • Cardiovascular • Immunology • Non-melanoma Skin Cancer • Oncology • Renal Disease

Comparison of Smoothened Inhibitors in Treating Advanced Basal Cell Carcinoma: Multidisciplinary Expert Opinions and Clinical Experiences (AAD 2026) - "Landmark studies ERIVANCE and BOLT facilitated FDA approval of vismodegib in 2012 and sonidegib in 2015, respectively, and provided valuable insights into SMOi efficacy, dosing, pharmacokinetics, and adverse effects (AE). These findings highlight the lack of uniformity, as well as familiarity, with the therapeutic options of SMOi for advanced BCC. Updating SMOi clinical guidelines and the creation of management strategies, including considerations for AE, dosing options, and drug switching, are needed to improve and standardize patient care."

Clinical • Metastases • Basal Cell Carcinoma • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer

Integrative pathway analysis of I-SPY2 HER2+ breast cancers reveals drug-repurposing opportunities (AACR 2026) - "Growth-factor/RTK bypass (PI3K/AKT) and DNA repair & oxidative stress defense were strongly upregulated in non-responders within BP-HER2, revealing actionable nodes involving PI3K/AKT (alpelisib, capivasertib), IGF1R (linsitinib), MET (crizotinib, capmatinib), and DNA repair (PARP inhibitors). Notably, metabolic rewiring and lipid homeostasis—targetable by vismodegib and sonidegib—were upregulated in non-responders across both BP subtypes, reflecting a shared metabolic vulnerability. Luminal biology-linked transcriptional programs may persist within the HER2+ BP-HER2 subtype and contribute to resistance... Luminal biology-linked transcriptional programs may persist within the HER2+ BP-HER2 subtype and contribute to resistance. BP-HER2 non-responders exhibit coordinated activation of RTK-bypass and DNA repair pathways, exposing therapeutic vulnerabilities targetable by existing agents. Furthermore, targeting lipid metabolic reprogramming alongside anti-HER2 therapy..."

Breast Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • HER-2

Sonidegib Therapy for Locally Advanced Basal Cell Carcinoma in an Elderly Patient: A Case Report. (PubMed, Clin Cosmet Investig Dermatol) - "Sonidegib (200 mg daily) was initiated, and a complete resolution was achieved after 5 months. No recurrence or significant adverse effects were observed 4 months post-treatment."

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

Sonidegib and Pembrolizumab in Treating Patients With Advanced Solid Tumors (clinicaltrials.gov) - P1 | N=36 | Active, not recruiting | Sponsor: Mayo Clinic | Trial completion date: Nov 2026 ➔ Jul 2027 | Trial primary completion date: Jan 2026 ➔ May 2025

Trial completion date • Trial primary completion date • Colorectal Cancer • Cutaneous Melanoma • Esophageal Cancer • Gastric Adenocarcinoma • Gastric Cancer • Gastroesophageal Junction Adenocarcinoma • Head and Neck Cancer • Lung Cancer • Melanoma • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck • Squamous Cell Skin Cancer • Urothelial Cancer • ALK • BRAF • EGFR • MSI • PD-L1

Final analysis of the multinational, post-authorisation study (NISSO) to assess the long-term safety and effectiveness of sonidegib in patients (pts) with locally advanced basal cell carcinoma (laBCC) (EADO 2026) - No abstract available

Clinical • Metastases • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

SATELLITE SYMPOSIUM 6: SCAR WARS: the laBCC Strikes Back – The Challenge of Recurrence in laBCC (EADO 2026) - "Sponsored by Sun Pharma Odomzo GOLD SPONSOR"

Case Report: PTCH1 splice-site mutation and sonidegib treatment in Gorlin-Goltz syndrome: clinical insights from a family case study. (PubMed, Front Med (Lausanne)) - "Dysgeusia and alopecia occurred with daily dosing, whereas every-other-day dosing was well tolerated. This case highlights the value of transcript-level functional assays for interpreting PTCH1 splice-site variants and supports individualized, toxicity-guided sonidegib scheduling in selected patients with Gorlin-Goltz syndrome."

Journal • Alopecia • Basal Cell Carcinoma • Immunology • Non-melanoma Skin Cancer • Oncology • PTCH1

Blocking SHH Pathway Reduces the in Vitro Malignant Behavior of Uterine Leiomyosarcoma via Pharmacological or Genetic Inhibition of SMO (ISGE 2026) - "Pharmacological inhibition employed two SMO antagonists (GDC-0449 and LDE225) at IC50-determined concentrations (GDC-0449: 40 µM; LDE225: 300 µM). SMO is overexpressed in uterine LMS and its pharmacologic or genetic inhibition attenuates Hedgehog pathway readouts and reduces malignant cell behaviors in vitro. These results support further evaluation of SMO-targeted strategies for uterine smooth muscle neoplasms."

IO biomarker • Preclinical • Leiomyosarcoma • Sarcoma • Solid Tumor • Uterine Leiomyosarcoma • BCL2 • BMP4 • CCND1 • GLI1 • GLI2 • PTCH1 • SMO

Collective synthesis of 1,2,4-trisubstituted, meta- and ortho-substituted arene bioisosteres from bicyclobutanes. (PubMed, Nat Chem) - "Moreover, the cycloadducts can be transformed into a diverse chemical space, including 1,5-disubstituted thiabicyclo[3.1.1]heptenes. Crystallographic analysis and a comparison of the pharmacokinetic properties, along with an evaluation of the biological activity of diflunisal, salicylanilide and the anticancer drug sonidegib, in relation to their 3D thia-BCHep analogues, demonstrate that the thia-BCHeps obtained can provide new surrogates for 1,2,4-trisubstituted, meta- and ortho-substituted benzene rings in drug discovery programmes."

Journal • Oncology

Shared clonal origin in a metastatic basal cell and scar-associated squamous cell carcinoma with therapeutic implications (DKK 2026) - "Pulmonary progression under sonidegib aligns with SMO-mediated resistance. Cemiplimab was reinitiated, and everolimus is under consideration based on the MTOR mutation."

IO biomarker • Metastases • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology • Squamous Cell Carcinoma • mTOR • MYCN • PTCH1

Increased Smoothened signaling promotes kidney fibrosis through inhibiting autophagy in fibroblasts. (PubMed, Kidney Int) - "Our study defines a prominent mechanism of myofibroblast activation and supplies a potential avenue for targeting fibroblast Smo to treat chronic kidney disease."

Journal • Cardiovascular • Chronic Kidney Disease • Fibrosis • Immunology • Nephrology • Renal Disease • Reperfusion Injury • mTOR

Pathological Insights into the Limitations of Clinical and Dermoscopic Evaluations in Monitoring Sonidegib Treatment Response for Locally Advanced Basal Cell Carcinoma: A Real-World Study. (PubMed, Dermatol Ther (Heidelb)) - "In this real-world Chinese laBCC cohort, sonidegib produced a clinically meaningful response with a favorable safety profile. However, clinical and dermoscopic assessments showed substantial false positives due to posttreatment changes; pathological biopsy remains essential to confirm tumor clearance. Advanced noninvasive imaging (e.g., reflectance confocal microscopy) may further improve monitoring. Prospective studies with longer follow-up are warranted."

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

UF-GI-015: AD HOC Trial: Artificial Intelligence-Based Drug Dosing In Hepatocellular Carcinoma (clinicaltrials.gov) - P2 | N=12 | Recruiting | Sponsor: University of Florida | Suspended ➔ Recruiting

Enrollment open • Hepatocellular Cancer • Oncology • Solid Tumor

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