Ameluge: Label Expansion for Broader Body Area Treatment
- A new clinical trial has brought Biofrontera one step closer to expanding the approved uses of its photodynamic therapy (PDT) treatment, Ameluz, for actinic keratosis (AK).
- Actinic keratoses are rough, scaly patches on the skin that develop from years of sun exposure.
- Currently, treatments for AK include topical creams like fluorouracil and imiquimod, as well as other PDT options.
A new clinical trial has brought Biofrontera one step closer to expanding the approved uses of its photodynamic therapy (PDT) treatment, Ameluz, for actinic keratosis (AK). Results from a Phase 3 study, , indicate the treatment is effective for AK lesions not only on the face and scalp – its currently approved areas – but also on the extremities, neck, and trunk.
Actinic keratoses are rough, scaly patches on the skin that develop from years of sun exposure. While often benign, these lesions are considered precancerous, with the potential to develop into squamous cell carcinoma. Early treatment is therefore considered the standard of care.
Expanding Treatment Options for Actinic Keratosis
Currently, treatments for AK include topical creams like fluorouracil and imiquimod, as well as other PDT options. However, these treatments aren’t always ideal. Topical creams can require prolonged application and may cause significant skin irritation. PDT, which involves applying a photosensitizing agent (like Ameluz) followed by exposure to a specific wavelength of light, often offers a shorter treatment duration and is administered by a clinician, providing more controlled application.
The Phase 3 trial focused on evaluating Ameluz PDT for AK on areas of the body where the skin tends to be thicker and lesions can be more resistant to traditional topical treatments – the extremities, neck, and trunk. According to Biofrontera, the successful completion of this trial is a necessary step before submitting a supplemental New Drug Application (sNDA) to the Food and Drug Administration (FDA).
How Ameluz Works
Ameluz utilizes aminolevulinic acid hydrochloride, a photosensitizing agent. When applied to AK lesions and then exposed to a specific light source, it selectively destroys the precancerous cells. The company specializes in dermatological treatments utilizing PDT.
Real-World Data and Label Expansion
The move to expand the label for Ameluz highlights a broader trend in the pharmaceutical industry: the increasing importance of real-world data (RWD) in supporting label expansions. Traditionally, regulatory approval and label specifications are based on data from randomized controlled trials (RCTs). However, RCTs don’t always reflect how a drug performs in everyday clinical practice.
RWD, collected from routine clinical practice, can provide valuable insights into a therapy’s effectiveness when delivered by general practitioners, used alongside other medications, and applied across diverse care settings. This is particularly important for understanding how a treatment performs in populations not fully represented in clinical trials, or in situations where strict trial protocols aren’t feasible.
Label expansion can significantly increase patient access to a treatment and strengthen its market position. Expanding the approved use of Ameluz to include the extremities, neck, and trunk addresses a large segment of the AK patient population, potentially offering a more effective treatment option for lesions in these areas.
Beyond Actinic Keratosis: Ameluz in Acne Research
Biofrontera is also exploring the potential of Ameluz for other dermatological conditions. A Phase 2b clinical trial evaluating Ameluz for the treatment of moderate to severe acne vulgaris is nearing completion, with top-line results expected in the first quarter of . The company plans to discuss the results with the FDA in the third quarter of to potentially advance the program to Phase 3 and seek label expansion for acne treatment.
Acne vulgaris affects more than 50 million people in the United States each year, representing a significant market valued at $5.7 billion in 2024. Current treatments for acne include topical agents, systemic antibiotics, hormonal therapies, and physical treatments, but many have limitations, including side effects and the development of antibiotic resistance. PDT with Ameluz could offer an alternative approach.
The Role of Real-World Evidence
The use of real-world evidence (RWE) is becoming increasingly recognized by regulatory agencies like the FDA in evaluating the safety and efficacy of medical products. While RCTs remain the gold standard, RWE can play a crucial role in supporting label expansions, particularly when RCTs are not feasible or practical. RWE can help demonstrate the effectiveness of a treatment in real-world settings, providing a more comprehensive understanding of its benefits and risks.
If the sNDA for expanded use of Ameluz is approved, it could improve the product’s competitive positioning against other topical therapies and PDT competitors. The success of the Phase 3 trial represents a significant milestone for Biofrontera as it seeks to broaden the application of its lead product and address a wider range of dermatological needs.