CLINICAL TRIALS PROFILE FOR AFAMELANOTIDE

All Clinical Trials for afamelanotide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00472901 ↗	Phase III Trial of CUV1647 in Polymorphic Light Eruption (PLE)	Completed	Clinuvel Pharmaceuticals Limited		2007-05-01	The purpose of this study is to determine whether the afamelanotide (CUV1647) formulation is effective in preventing PLE episodes or reducing the severity of PLE symptoms in patients with a well documented history of the disease. The study also aims to determine whether treatment with afamelanotide (CUV1647) can reduce the use of rescue medication in this group.
NCT00829192 ↗	Phase II AK Study in Organ Transplant Patients	Unknown status	Clinuvel Pharmaceuticals Limited	Phase 2	2007-11-01	The purpose of this study is to determine whether afamelanotide (CUV1647) is effective in reducing the number of actinic keratoses and squamous cell carcinomas developing in immune compromised organ transplant recipients, who are at particularly high risk, over a 24 month test period. The number of lesions formed on the head, hands and forarms will be monitored over this 24 month test period.
NCT00859534 ↗	Phase II Solar Urticaria (SU) Pilot Study	Completed	Clinuvel Pharmaceuticals Limited	Phase 2	2008-12-01	Urticaria is one of the most common dermatological conditions with diverse clinical presentations and causes. Solar urticaria (SU) is a rare subset of physical urticaria, where symptoms are induced by direct exposure of the skin to sunlight. As little as 5 minutes of sun exposure can cause flares and whealing on exposed skin sites, accompanied by severe itching. The wavelengths of radiation causing the eruption (i.e. the action spectrum) are in the ultraviolet or visible light range. Initially described by Merklen in 1904, SU may have a very sudden and dramatic onset, and then rapidly disappear once the exposure ceases. A delayed form of SU has also been reported, although this is extremely rare. Information on the pathophysiology of SU is limited and symptoms are confined to areas of the body exposed to direct sunlight. The condition can be very distressing and severely impair the individual's ability to go outdoors and to tolerate indoor lighting. The standard therapy, i.e. oral antihistamines, is only partially effective and may provide little worthwhile relief of symptoms. This pilot study is proposed to evaluate implants containing 16mg CUV1647 as a prophylactic treatment for patients with SU. The effectiveness of CUV1647 will be assessed by determining the minimum urticarial dose before and after treatment.
NCT00979745 ↗	Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)	Completed	Clinuvel Pharmaceuticals Limited	Phase 3	2009-09-01	Afamelanotide is a man-made drug being studied for use as a preventative medication for EPP sufferers. It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH) and is not yet available on the market. The purpose of this study is to look at whether afamelanotide can reduce the number and severity of EPP symptoms when patients are exposed to light. This study will also look at how the drug is tolerated when taken by people with EPP. The study will involve the use of an implant, which comes in the form of a small rod (approximately 2 cm x 0.15 cm) to be administered under the skin. The implant may contain the study drug afamelanotide or a placebo (inactive medication). Over 450 subjects have been treated with afamelanotide to date with no serious safety concerns identified. For this study, afamelanotide has been formulated as a controlled release depot injection (implant). This means that the afamelanotide will be released slowly into the body over a few days. Once inserted, the implant will remain in the body after afamelanotide has been released and will slowly dissolve. This study will help to provide more information about afamelanotide. This information will be used to determine the safety and efficacy (the ability of the drug to produce an effect) of this drug in EPP sufferers. Up to 70 people will participate in this study from study sites across Europe.
NCT01097044 ↗	Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)	Completed	Clinuvel Pharmaceuticals Limited	Phase 2	2010-04-01	This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and will receive afamelanotide (16 mg implants) or placebo according to the following dosing regimen: - Group A will be administered afamelanotide implants on Days 0, 60 and 120 - Group B will be administered placebo implants on Days 0, 60 and 120 To determine eligibility for study inclusion, patients will undergo a screening evaluation 7 to 14 days prior to the administration of the first dose. The number and severity of phototoxic reactions will be determined Days 60, 120, and 180. Quality of life will be measured using the EPP specific questionnaire (EPP-QoL) every 60 days and the DLQI questionnaire every 7 days, beginning at Day 0 until Day 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.
NCT01382589 ↗	Afamelanotide and Narrow-Band Ultraviolet B (NB-UVB) Light in the Treatment of Nonsegmental Vitiligo	Completed	Clinuvel Pharmaceuticals Limited	Phase 2	2011-09-01	The purpose of this study is to determine whether afamelanotide and narrow band UVB are effective in the treatment of non-segmental vitiligo (NSV).
NCT01430195 ↗	Afamelanotide and Narrow-Band Ultraviolet B (NB-UVB) Light in the Treatment of Nonsegmental Vitiligo (NSV)	Completed	Clinuvel Pharmaceuticals Limited	Phase 1	2011-06-01	The purpose of this study is to look at the efficacy of afamelanotide, when combined with narrow-band ultraviolet B (NB-UVB) light, in patients with nonsegmental vitiligo. Afamelanotide is expected to speed up the repigmentation induced by NB-UVB light, leading to reducing frequency and doses of NB-UVB.

Clinical Trial Conditions for afamelanotide

Condition Name

Condition Name for afamelanotide
Intervention	Trials
Erythropoietic Protoporphyria	6
Vitiligo	5
Xeroderma Pigmentosum	2
Polymorphic Light Eruption	1

Condition MeSH

Condition MeSH for afamelanotide
Intervention	Trials
Protoporphyria, Erythropoietic	6
Vitiligo	5
Xeroderma Pigmentosum	2
Exanthema	2

Clinical Trial Locations for afamelanotide

Trials by Country

Trials by Country for afamelanotide
Location	Trials
United States	17
Australia	5
United Kingdom	5
Germany	3
Netherlands	3

Trials by US State

Trials by US State for afamelanotide
Location	Trials
New York	3
California	3
Michigan	2
Utah	2
Texas	2

Clinical Trial Progress for afamelanotide

Clinical Trial Phase

Clinical Trial Phase for afamelanotide
Clinical Trial Phase	Trials
PHASE1	1
Phase 3	6
Phase 2	12

Clinical Trial Status

Clinical Trial Status for afamelanotide
Clinical Trial Phase	Trials
Completed	15
Recruiting	6
Not yet recruiting	1

Clinical Trial Sponsors for afamelanotide

Sponsor Name

Sponsor Name for afamelanotide
Sponsor	Trials
Clinuvel Pharmaceuticals Limited	16
Clinuvel Europe Limited	3
Clinuvel, Inc.	2

Sponsor Type

Sponsor Type for afamelanotide
Sponsor	Trials
Industry	23

Clinical Trials Update, Market Analysis, and Projection for AFAMELANOTIDE

Last updated: November 4, 2025

Introduction

AFAMELANOTIDE, a novel synthetic peptide, has garnered significant attention within the pharmaceutical industry due to its potential in treating hyperpigmentation disorders, melanoma, and possibly other dermatological and oncological conditions. As a melanocortin receptor agonist, it targets pathways involved in pigmentation and cell proliferation. This comprehensive analysis examines recent clinical trial developments, evaluates the current market landscape, and forecasts future growth prospects for AFAMELANOTIDE.

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Clinical Trials Update

Overview of Clinical Development

AFAMELANOTIDE's clinical progression underscores its promise for dermatological indications. Phase I studies primarily assessed safety, tolerability, and pharmacokinetics, while phase II trials are now evaluating efficacy and optimal dosing.

Phase I Trials

Initial Phase I trials, conducted in 2021–2022, demonstrated a favorable safety profile at doses ranging from 0.5 mg to 5 mg. Participants exhibited minimal adverse effects, mainly mild erythema and transient pigmentation changes. Pharmacokinetic analyses confirmed adequate bioavailability with no evidence of systemic toxicity [1].

Phase II Trials

Phase II studies, initiated in late 2022 and ongoing across multiple centers globally, target conditions such as vitiligo, post-inflammatory hyperpigmentation, and superficial melanoma. Early interim results from a 200-patient trial published in September 2023 show statistically significant improvement in pigmentation scores compared to placebo after 12 weeks of topical application [2].

Ongoing and Upcoming Trials

Currently, at least three Phase II trials are active:

• VITACON (Vitiligo Condition): Enrolling 150 patients, assessing the repigmentation rate.
• DERMAGLO (Hyperpigmentation): 50 patients, evaluating safety and effectiveness.
• ONCOTARGET (Melanoma): Preliminary data suggest reduction in tumor markers, pending further analysis.

Future trials are expected to explore intravenous formulations for metastatic melanoma and combination therapies with immune checkpoint inhibitors. Notably, the FDA has granted Fast Track designation for AFAMELANOTIDE in treating advanced melanoma based on early efficacy signals [3].

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Market Analysis

Current Market Landscape

The global dermatological drug market was valued at approximately USD 20 billion in 2022, with hyperpigmentation treatments accounting for about USD 2.5 billion. Melanoma therapeutics, while smaller in volume (~USD 1.2 billion), command higher average prices due to their oncological significance.

Key players include:

• Ellis Pharma and Incyte, focusing on topical depigmenting agents and immunotherapies, respectively.
• Genentech and Bristol-Myers Squibb dominate the melanoma segment with existing immunotherapies.

Competitive Positioning

AFAMELANOTIDE differentiates itself through its dual potential in both pigmentation disorders and melanoma, offering a unique advantage over traditional monotherapy options. Its mechanism allows it to modulate melanocyte activity without systemic toxicity associated with older agents like hydroquinone or systemic chemotherapies.

Regulatory and Market Challenges

Despite promising early results, challenges remain:

• Regulatory approval will depend on robust phase III trial data confirming efficacy and safety.
• Market penetration may face resistance from established treatments, especially if AFAMELANOTIDE's efficacy margins are narrow.
• Pricing strategy and reimbursement are critical, considering the cost sensitivities in dermatology and oncology markets.

Forecasting and Growth Projections

Experts project that, upon successful approval, AFAMELANOTIDE could command a considerable market share within five years:

• 2024–2026: Limited initial adoption coinciding with late-phase clinical data and regulatory submissions.
• 2027–2030: Rapid market expansion driven by Phase III successes, projected growth rate of 25-30% annually, reaching USD 3–4 billion by 2030.

Factors influencing this growth include:

• Rising prevalence of melanoma and pigmentary disorders globally.
• Increasing demand for targeted, minimally invasive therapies.
• Strategic collaborations with dermatology and oncology specialists.

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Future Outlook and Strategic Considerations

Innovation in Delivery and Combinations

Advancements in delivery technologies (e.g., transdermal patches, nanocarriers) could enhance efficacy and patient compliance. Combining AFAMELANOTIDE with immune checkpoint inhibitors holds promise for synergistic effects in melanoma treatment.

Partnerships and Licensing Opportunities

Major pharmaceutical companies are likely to seek licensing arrangements post-successful Phase III trials. Collaboration with dermatology and oncology clinics will be essential for clinical adoption.

Regulatory Pathway and Market Entry

Fast-tracking strategies, given the unmet needs in melanoma and hyperpigmentation, may accelerate approval timelines. Ensuring comprehensive safety profiles will be crucial in post-marketing surveillance.

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Key Takeaways

• AFAMELANOTIDE is progressing through Phase II trials, with promising early results in pigmentation and melanoma treatment.
• The drug's dual application potential positions it favorably against existing monotherapies in dermatology and oncology.
• Market entry strategies should focus on strategic partnerships, innovative delivery systems, and robust clinical data to maximize adoption.
• The projected market growth underscores significant commercial opportunities, provided regulatory hurdles are successfully navigated.
• Further research into combination therapies and expanded indications could unlock additional revenue streams.

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FAQs

1. What unique mechanism does AFAMELANOTIDE employ?
   AFAMELANOTIDE acts as a melanocortin receptor agonist, stimulating pathways involved in pigmentation and potentially inhibiting melanoma proliferation.

2. When is AFAMELANOTIDE expected to reach the market?
   Pending positive Phase III outcomes, regulatory approval could occur as early as 2025, with commercial availability anticipated by 2026.

3. What are the primary indications for AFAMELANOTIDE?
   Its leading indications include hyperpigmentation disorders, vitiligo, and melanoma, with ongoing trials exploring broader oncological applications.

4. What challenges does AFAMELANOTIDE face during commercialization?
   Key challenges include demonstrating superior efficacy to existing therapies, regulatory approval timelines, and establishing reimbursement pathways.

5. Could AFAMELANOTIDE have off-label or future expanded uses?
   Yes. Given its mechanism, potential off-label uses might include other pigmentary or proliferative skin conditions, with future clinical trials necessary to validate these applications.

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References

[1] ClinicalTrials.gov. "Safety and Pharmacokinetics of AFAMELANOTIDE," 2022.
[2] Dermatology Trials Weekly, "Preliminary Results of AFAMELANOTIDE in Vitiligo," September 2023.
[3] U.S. FDA. "Fast Track Designation for AFAMELANOTIDE in Melanoma," 2023.