Basal Cell Carcinoma Medication

Updated: Feb 14, 2022
  • Author: Robert S Bader, MD; Chief Editor: Mark S Granick, MD, FACS  more...
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Medication Summary

Medications used for the treatment of basal cell carcinoma (BCC) include the following:

  • Topical antineoplastic agents (eg, 5-fluorouracil, imiquimod)
  • The photosensitizing agent methyl aminolevulinate cream
  • The acetylenic retinoid tazarotene
  • Hedgehog pathway inhibitors (HHIs)
  • A programmed death–1 (PD-1) inhibitor

Antineoplastic Agents

Class Summary

The most common chemotherapeutic agent used in superficial basal cell carcinoma is topical 5-fluorouracil.

Fluorouracil topical (Efudex)

5-Fluorouracil topical 5% cream or solution is used topically for the management of superficial BCC. It interferes with DNA synthesis by blocking methylation of deoxyuridylic acid and inhibiting thymidylate synthetase and, subsequently, cell proliferation.

Interferon alfa-2b (Intron-A)

Interferon alfa-2b is a protein product manufactured with recombinant DNA technology. The mechanism of antitumor activity is not clearly understood; however, direct antiproliferative effects against malignant cells and modulation of host immune response may be important.

As an investigational drug for nodular BCC, interferon alfa-2b was used in a randomized, placebo-controlled multicenter study with 172 patients. Intralesional injections of 1.5 million U administered 3 times/wk for 3 wk yielded an 86% complete-response rate, compared with 29% for placebo. A similar study did not show efficacy for morpheaform or aggressive BCCs.


Antineoplastics, Hedgehog Pathway Inhibitor

Class Summary

The Hedgehog signaling pathway is important in embryogenesis, but in adults, it is mostly inactive. Signaling is relayed by key proteins including Smoothened homologue (SMO). Hedgehog ligand-expressing cancerous epithelial cells that are activated by the hedgehog signaling pathway may cause growth-promotion.

Vismodegib (Erivedge)

Vismodegib is a Hedgehog (Hh) pathway inhibitor. Vismodegib binds to and inhibits SMO, a transmembrane protein involved in Hedgehog signal transduction. This agent is indicated for treatment of adults with metastatic basal cell carcinoma or with locally advanced basal cell carcinoma that has recurred following surgery, or those who are not candidates for surgery or radiation.

Sonidegib (Odomzo)

Sonidegib binds to and inhibits SMO, a transmembrane protein involved in Hedgehog signal transduction. Indicated for adults with locally advanced basal cell carcinoma that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.


PD-1/PD-L1 Inhibitors

Cemiplimab (Cemiplimab-rwlc, Libtayo)

Recombinant human immunoglobulin G4 monoclonal antibody binds to PD-1 and blocks its interaction with PD-L1 and PD-L2, releasing PD-1 pathway-mediated inhibition of the immune response, including the anti-tumor immune response. It is indicated for locally advanced BCC and granted accelerated approval for metastatic BCC previously treated with a HHI or for whom a HHI is not appropriate.


Photosensitizing Agent, Topical

Class Summary

Photodynamic therapy (PDT) for basal cell carcinomas is the process of using specific wavelengths of light to photoexcite porphyrins that have been applied to neoplastic and preneoplastic cells. This increased energy is rapidly absorbed by adjacent tissue oxygen, causing the formation of singlet oxygen radicals. These radicals rapidly react with adjacent tissue and destroy it.

Methyl aminolevulinate (Metvixia)

Methyl aminolevulinate cream is a porphyrin precursor used in combination with narrow-band, red-light illumination for nonhyperkeratotic, nonpigmented actinic keratoses. When used with photodynamic therapy, the accumulation of photoactive porphyrins produces a photodynamic reaction that results in a cytotoxic process dependent upon the simultaneous presence of oxygen.


Keratolytic Agents

Class Summary

These agents decrease cohesiveness of abnormal hyperproliferative keratinocytes and may reduce potential for malignant degeneration. It modulates keratinocyte differentiation.

Tazarotene (Avage, Fabior, Tazorac)

Tazarotene is a retinoid prodrug with an active metabolite that modulates differentiation and proliferation of epithelial tissue; it also may have anti-inflammatory and immunomodulatory properties. It is not approved by the US Food and Drug Administration for treating basal cell carcinoma.


Topical Skin Products

Class Summary

Products that induce cytokines, including interferon alpha, have been shown to be effective.

Imiquimod (Aldara)

The precise mechanism of imiquimod for superficial BCC is unknown. It may increase tumor infiltration by lymphocytes, dendritic cells, and macrophages. It is indicated for biopsy-confirmed primary nonfacial superficial BCC in adults with normal immune systems. Additionally, tumors must not exceed 2 cm in diameter on certain areas of the body. Imiquimod is indicated only when surgical methods are not appropriate.