FDA Approves Aminolevulinic Acid Hydrochloride Plus PDT for Basal Cell Carcinoma

The FDA has approved aminolevulinic acid hydrochloride (Ameluz) topical gel, in combination with photodynamic therapy (PDT) using the BF-RhodoLED lamp series for the treatment of adult patients with superficial basal cell carcinoma.

The regulatory decision was backed by data from a multicenter, randomized, double-blind, vehicle-controlled phase 3 trial (NCT03573401), which showed that 66% of patients treated with aminolevulinic acid hydrochloride plus PDT (n = 145) experienced both clinical and histological complete response (CR) of a main target lesion 12 weeks after the start of the last PDT cycle compared with 5% for patients treated with placebo plus PDT (n = 45). Additionally, 76% of patients treated in the experimental arm achieved histological clearance of the main target lesion vs 19% of patients in the placebo arm. The respectiv rates of complete clinical clearance of all target lesions were 83% vs 21%.

"FDA approval of [aminolevulinic acid hydrochloride] for superficial basal cell carcinoma is a defining milestone for Biofrontera. Ameluz is now the first and only photodynamic therapy approved in the United States to treat a skin cancer, which sets our platform apart in medical dermatology and gives dermatologists an effective, non-surgical option for a very common tumor,” Hermann Luebbert, chief executive officer and chairman of Biofrontera, stated in a news release. “This approval reflects years of disciplined investment in the clinical development of Ameluz, and the latest stage in the ongoing expansion of our PDT platform.”

How was aminolevulinic acid hydrochloride investigated in the phase 3 trial?

The multicenter, randomized, double-blind, placebo-controlled trial enrolled 187 adult patients with at least 1 histologically confirmed superficial basal cell carcinoma lesion on the face/forehead, bald scalp, extremities, and/or neck trunk.² Each enrolled patient had 1 lesion defined as the primary target lesion.

In both arms, all patients received at least 1 chycle of PDT, comprising 2 PDT sessions 7 to 14 days apart. During each session, patients also had their primary lesion covered in aminolevulinic acid hydrochloride or placebo for 3 hours prior to the area being illuminated with the BF-RhodoLED lamp.

The rate of patients with composite clinical and histological CR of the primary target lesion 12 weeks after the start of the last PDT cycle served as the trial’s primary end point.

What safety data were reported for aminolevulinic acid hydrochloride?

In the aminolevulinic acid hydrochloride arm, all patients (n = 145) experienced at least 1 adverse effect (AE) at the application site.

The most common any-grade AEs reported in at least 1% of patients in the experimental arm included pain (aminolevulinic acid hydrochloride, 95%; placebo, 29%), erythema (69%; 36%), pruritus (50%; 26%), edema (29%; 12%), paraesthesia (24%; 12%), scab (21%; 5%), induration (17%; 7%), exfoliation (17%; 14%), discoloration (9%; 2%), vesicules (8%; 2%), dysaesthesia (2%; 0%), hemorrhage (2%; 0%), dryness (1%; 0%), hyperesthesia (1%; 0%), and swelling (1%; 0%).

"For many patients with superficial basal cell carcinoma—those with multiple lesions, lesions in cosmetically sensitive locations, or for whom surgery would not be preferred—treatment options have been limited,” M. Shane Chapman, MD, MBA, a board certified dermatologist and professor and founding chair of the Department of Dermatology at Dartmouth Hitchcock Medical Center and the Dartmouth Geisel School of Medicine, added in a news release.¹ “This FDA approval for [aminolevulinic acid hydrochloride] and the RhodoLED gives us a rigorously studied, non-surgical 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."

References

1. Biofrontera announces FDA approval of Ameluz red light PDT for the treatment of superficial basal cell carcinoma. News release. Biofrontera. September 14, 2026. Accessed September 14, 2026. https://investors.biofrontera-us.com/full-news/?qm-storyId=6773571413757050
2. Ameluz. Prescribing information. Updated September 2026. Accessed September 14, 2026. https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/208081s040lbl.pdf