Today, the U.S. Food and Drug Administration (FDA) approved the supplemental New Drug Application for aminolevulinic acid hydrochloride topical gel, 10% (Ameluz), in combination with photodynamic therapy (PDT) using the BF-RhodoLED lamp series, for the treatment of superficial basal cell carcinoma (BCC) in adult patients.
With this approval, aminolevulinic acid hydrochloride becomes the first and only PDT drug approved by the FDA for the treatment of a skin cancer, and the only topical PDT in the United States indicated to treat both a precancerous skin condition (actinic keratosis) and a skin cancer.
BCC is the most common skin cancer in the United States, with approximately 3.6 million cases diagnosed annually, according to the Skin Cancer Foundation. Published estimates suggest that the superficial subtype accounts for approximately 15% to 20% of BCC cases, or an estimated 540,000 to 720,000 cases annually in the United States. Superficial BCC lesions can measure up to several centimeters in diameter and are frequently located on the trunk and extremities and often present as multiple lesions, for which nonsurgical, tissue-sparing treatment options are limited.
Phase III Trial
The FDA approval is supported by a multicenter, randomized, double-blind, vehicle-controlled phase III trial in 187 adults with histologically confirmed superficial BCC (ClinicalTrials.gov identifier NCT03573401). The primary endpoint was a composite of both clinical and histologic complete response of a main target lesion 12 weeks after the start of the last PDT cycle. This target was achieved in 66% of subjects treated with aminolevulinic acid hydrochloride PDT (n = 95 of 145) compared with 5% of subjects treated with placebo-PDT (n = 2 of 42). Histologic clearance of the main target lesion was seen in 76% of aminolevulinic acid hydrochloride PDT–treated patients vs 19% of patients treated with placebo-PDT, and 83% of aminolevulinic acid hydrochloride PDT–treated subjects achieved complete clinical clearance of all target lesions compared with 21% for placebo-PDT.
The most common adverse reactions were application-site reactions consistent with the known safety profile of aminolevulinic acid hydrochloride PDT.
M. Shane Chapman, MD, MBA, Professor and Founding Chair of the Department of Dermatology at Dartmouth-Hitchcock Medical Center and the Dartmouth Geisel School of Medicine, and a key contributor to the phase III study, commented, "For many patients with superficial BCC—those with multiple lesions, lesions in cosmetically sensitive locations, or for whom surgery would not be preferred—treatment options have been limited. This FDA approval ... gives us a rigorously studied, nonsurgical option that we can deliver in our office. As someone who has worked with PDT for many years, I see this as a meaningful expansion in the use of this treatment."
