Sonidegib for the treatment of advanced basal cell carcinoma

References

• Goldenberg G, Karagiannis T, Palmer JB, et al. Incidence and prevalence of basal cell carcinoma (BCC) and locally advanced BCC (LABCC) in a large commercially insured population in the United States: a retrospective cohort study. J Am Acad Dermatol. 2016 Jul 26. doi:10.1016/j.jaad.2016.06.020. [Epub ahead of print].
   PubMedGoogle Scholar
• Arits AH, Mosterd K, Essers BA, et al. Photodynamic therapy versus topical imiquimod versus topical fluorouracil for treatment of superficial basal-cell carcinoma: a single blind, non-inferiority, randomised controlled trial. Lancet Oncol. 2013 Jun;14(7):647–654.
   PubMed Web of Science ®Google Scholar
• Roozeboom MH, Arits AH, Mosterd K, et al. Three year follow-up results of photodynamic therapy versus imiquimod versus fluorouracil for treatment of superficial basal cell carcinoma: a single blind, non-inferiority, randomized controlled trial. J Invest Dermatol. 2016 Aug;136(8):1568–1574.
   PubMed Web of Science ®Google Scholar
• Zagrodnik B, Kempf W, Seifert B, et al. Superficial radiotherapy for patients with basal cell carcinoma: recurrence rates, histologic subtypes, and expression of p53 and Bcl-2. Cancer. 2003 Dec 15;98(12):2708–2714.
   PubMed Web of Science ®Google Scholar
• Lear JT, Corner C, Dziewulski P, et al. Challenges and new horizons in the management of advanced basal cell carcinoma: a UK perspective. Br J Cancer. 2014 Oct 14;111(8):1476–1481.
   PubMed Web of Science ®Google Scholar
• Dreier J, Cheng PF, Bogdan Alleman I, et al. Basal cell carcinomas in a tertiary referral centre: a systematic analysis. Br J Dermatol. 2014 Nov;171(5):1066–1072.
   PubMed Web of Science ®Google Scholar
• Amici JM, Battistella M, Beylot-Barry M, et al. Defining and recognising locally advanced basal cell carcinoma. Eur J Dermatol. 2015 Dec 1;25(6):586–594.
   PubMed Web of Science ®Google Scholar
• Nägeli MC, Dummer R. Vismodegib (Erivedge). Schweiz Med Forum. 2014;14(13):284–286.
   Google Scholar
• Paver K, Poyzer K, Burry N, et al. Letter: the incidence of basal cell carcinoma and their metastases in Australia and New Zealand. Australas J Dermatol. 1973 Apr;14(1):53.
   PubMedGoogle Scholar
• Ting PT, Kasper R, Arlette JP. Metastatic basal cell carcinoma: report of two cases and literature review. J Cutan Med Surg. 2005 Jan;9(1):10–15.
   PubMed Web of Science ®Google Scholar
• McCusker M, Basset-Seguin N, Dummer R, et al. Metastatic basal cell carcinoma: prognosis dependent on anatomic site and spread of disease. Eur J Cancer. 2014 Mar;50(4):774–783.
   PubMed Web of Science ®Google Scholar
• Epstein EH. Basal cell carcinomas: attack of the hedgehog. Nat Rev Cancer. 2008 Oct;8(10):743–754.
   PubMed Web of Science ®Google Scholar
• Sekulic A, Migden MR, Oro AE, et al. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. N Engl J Med. 2012 Jun 7;366(23):2171–2179.
   PubMed Web of Science ®Google Scholar
• Gailani MR, Bale SJ, Leffell DJ, et al. Developmental defects in Gorlin syndrome related to a putative tumor suppressor gene on chromosome 9. Cell. 1992 Apr 3;69(1):111–117.
   PubMed Web of Science ®Google Scholar
• Hahn H, Wicking C, Zaphiropoulous PG, et al. Mutations of the human homolog of Drosophila patched in the nevoid basal cell carcinoma syndrome. Cell. 1996 Jun 14;85(6):841–851.
   PubMed Web of Science ®Google Scholar
• Johnson RL, Rothman AL, Xie J, et al. Human homolog of patched, a candidate gene for the basal cell nevus syndrome. Science. 1996 Jun 14;272(5268):1668–1671.
   PubMed Web of Science ®Google Scholar
• Epstein EH Jr. Mommy – where do tumors come from? J Clin Invest. 2011 May;121(5):1681–1683.
   PubMed Web of Science ®Google Scholar
• Hutchin ME, Kariapper MS, Grachtchouk M, et al. Sustained Hedgehog signaling is required for basal cell carcinoma proliferation and survival: conditional skin tumorigenesis recapitulates the hair growth cycle. Genes Dev. 2005 Jan 15;19(2):214–223.
   PubMed Web of Science ®Google Scholar
• Smith MJ, Beetz C, Williams SG, et al. Germline mutations in SUFU cause Gorlin syndrome-associated childhood medulloblastoma and redefine the risk associated with PTCH1 mutations. J Clin Oncol. 2014 Dec 20;32(36):4155–4161.
   PubMed Web of Science ®Google Scholar
• Otsuka A, Levesque MP, Dummer R, et al. Hedgehog signaling in basal cell carcinoma. J Dermatol Sci. 2015 May;78(2):95–100.
   PubMed Web of Science ®Google Scholar
• Di Magno L, Coni S, Di Marcotullio L, et al. Digging a hole under Hedgehog: downstream inhibition as an emerging anticancer strategy. Biochim Biophys Acta. 2015 Aug;1856(1):62–72.
   PubMed Web of Science ®Google Scholar
• Stone DM, Murone M, Luoh S, et al. Characterization of the human suppressor of fused, a negative regulator of the zinc-finger transcription factor Gli. J Cell Sci. 1999 Dec;112(Pt 23):4437–4448.
   PubMed Web of Science ®Google Scholar
• Bale AE. Sheep, lilies and human genetics. Nature. 2000 Aug 31;406(6799):944–945.
   PubMed Web of Science ®Google Scholar
• Dreier J, Dummer R, Felderer L, et al. Emerging drugs and combination strategies for basal cell carcinoma. Expert Opin Emerg Drugs. 2014 Sep;19(3):353–365.
   PubMed Web of Science ®Google Scholar
• Tang JY, Mackay-Wiggan JM, Aszterbaum M, et al. Inhibiting the hedgehog pathway in patients with the basal-cell nevus syndrome. N Engl J Med. 2012 Jun 7;366(23):2180–2188.
   PubMed Web of Science ®Google Scholar
• Pan S, Wu X, Jiang J, et al. Discovery of nvp-lde225, a potent and selective smoothened antagonist. acs med chem lett. 2010 jun 10;1(3):130–134.
   PubMed Web of Science ®Google Scholar
• Rodon J, Tawbi HA, Thomas AL, et al. A phase I, multicenter, open-label, first-in-human, dose-escalation study of the oral smoothened inhibitor sonidegib (LDE225) in patients with advanced solid tumors. Clin Cancer Res. 2014 Apr 1;20(7):1900–1909.
   PubMed Web of Science ®Google Scholar
• Goel V, Hurh E, Stein A, et al. Population pharmacokinetics of sonidegib (LDE225), an oral inhibitor of hedgehog pathway signaling, in healthy subjects and in patients with advanced solid tumors. Cancer Chemother Pharmacol. 2016 Apr;77(4):745–755.
   PubMed Web of Science ®Google Scholar
• Zollinger M, Lozac’h F, Hurh E, et al. Absorption, distribution, metabolism, and excretion (ADME) of (1)(4)C-sonidegib (LDE225) in healthy volunteers. Cancer Chemother Pharmacol. 2014 Jul;74(1):63–75.
   PubMed Web of Science ®Google Scholar
• Migden MR, Guminski A, Gutzmer R, et al. Treatment with two different doses of sonidegib in patients with locally advanced or metastatic basal cell carcinoma (BOLT): a multicentre, randomised, double-blind phase 2 trial. Lancet Oncol. 2015 Jun;16(6):716–728.
   PubMed Web of Science ®Google Scholar
• Dummer R, Migden MR, Guminski A, et al. Efficacy and safety of sonidegib in patients with locally advanced or metastatic basal cell carcinoma: bolt 30-month analysis. 2016 ASCO Annual Meeting; 2016; Chicago (IL)
   Google Scholar
• Dummer R, Guminski A, Gutzmer R, et al. The 12-month analysis from basal cell carcinoma outcomes with lde225 treatment (BOLT): a phase II, randomized, double-blind study of sonidegib in patients with advanced basal cell carcinoma. J Am Acad Dermatol. 2016 Jul;75(1):113–125.e5.
   PubMed Web of Science ®Google Scholar
• Lear JMM, Guminski a, dirix l, et al. Efficacy and safety of sonidegib in patients with nevoid basal cell carcinoma syndrome. 11th EADO Congress and 8th World Meeting of Interdisciplinary Melanoma/Skin Centers; 2015; Marseille.
   Google Scholar
• Atwood SX, Sarin KY, Whitson RJ, et al. Smoothened variants explain the majority of drug resistance in basal cell carcinoma. Cancer Cell. 2015 Mar 9;27(3):342–353.
   PubMed Web of Science ®Google Scholar
• Sharpe HJ, Pau G, Dijkgraaf GJ, et al. Genomic analysis of smoothened inhibitor resistance in basal cell carcinoma. Cancer Cell. 2015 Mar 9;27(3):327–341.
   PubMed Web of Science ®Google Scholar
• Danial C, Sarin KY, Oro AE, et al. An investigator-initiated open-label trial of sonidegib in advanced basal cell carcinoma patients resistant to vismodegib. Clin Cancer Res. 2016 mar 15;22(6):1325–1329.
   PubMed Web of Science ®Google Scholar
• Sekulic A, Migden MR, Lewis K, et al. Pivotal ERIVANCE basal cell carcinoma (BCC) study: 12-month update of efficacy and safety of vismodegib in advanced BCC. J Am Acad Dermatol. 2015 Jun;72(6):1021–6e8.
   PubMed Web of Science ®Google Scholar
• EMA: summary of opinion Odomzo [Internet]. 2015 [cited 2016]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion_-_Initial_authorisation/human/002839/WC500188762.pdf
   Google Scholar
• Swissmedic authorisation of medical product [Internet]. 2015 [ cited; Available from: https://www.swissmedic.ch/zulassungen/00153/00189/00200/02858/index.html?lang=de
   Google Scholar
• FDA approves new treatment for most common form of advanced skin cancer [Internet]. 2015 [cited 2016 May 20]. Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm455862.htm
   Google Scholar
• FDA: highlights of prescribing information Odomzo [Internet]. 2015 [cited 2016 May 20]. Available from: http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/205266s000lbl.pdf
   Google Scholar
• Otsuka A, Dreier J, Cheng PF, et al. Hedgehog pathway inhibitors promote adaptive immune responses in basal cell carcinoma. Clin Cancer Res. 2015 Mar 15;21(6):1289–1297.
   PubMed Web of Science ®Google Scholar