Scenesse (afamelanotide) / Clinuvel - LARVOL DELTA

Afamelanotide: A Novel Promising Treatment for Vitiligo - Case Studies from the Randomized CUV105 Clinical Trial and its Extension Phase (EADV 2026) - No abstract available

Case study • Clinical • Dermatology • Immunology • Vitiligo

Acute impact of afamelanotide on UVR erythemal and melanogenic responses in healthy humans in vivo: an exploratory study. (PubMed, Photochem Photobiol Sci) - "Melanin density (reflectance at 420 and 400 nm) increased (mean overall increase 2.4% to 2.9%, P = 0.001), while melanin staining was unaltered. The demonstrated protection against acute UVR-induced erythema shortly after afamelanotide application supports that anti-inflammatory effects observed in vitro may operate in vivo, warranting further investigation in acute UVR-induced inflammatory conditions."

Journal • Dermatology • Genetic Disorders • Inflammation • Metabolic Disorders

A Review of Randomized Controlled Trials for Vitiligo Therapies Published Between 2013 and 2023. (PubMed, Cureus) - "We further narrowed our focus to studies that could be aggregated into the intervention categories: phototherapy and systemic combination therapy (n=10), topical and topical combination therapy (n=15), and systemic monotherapy (n=5).  Treated versus control subjects showed statistically improved VASI after the following interventions were added to phototherapy: oral psoralen, oral minipulsed prednisone, implanted afamelanotide, topical ethyl vanillate, topical bimatoprost, and oral vitamins A and E. Effective topical therapies were ruxolitinib, calcipotriol and betamethasone, tacrolimus and mometasone, and microdermabrasion and tacrolimus. None of the systemic monotherapies was superior to their corresponding comparator."

Journal • Review • Aesthetic Medicine • Dermatology • Immunology • Vitiligo

SCENESSE approved for EPP in Canada (Yahoo Finance) - "The NOC acknowledges the compliance of the SCENESSE new drug submission with Section C.08.002 and C.08.005 of the Canadian Food and Drug Regulations and grants CLINUVEL the right to market SCENESSE in Canada."

Canada approval • Dermatology • Genetic Disorders

A systematic review of case series and clinical trials investigating systemic oral or injectable therapies for the treatment of vitiligo (EADV-Sp 2026) - "OMP, Methotrexate, Azathioprine, Cyclosporine, Mycophenolate mofetil, Simvastatin, Apremilast, Minocycline, Afamelanotide, Tofacitinib, Baricitinib, Antioxidants, and oral/injectable corticosteroids are effective treatment methods. However, oral zinc and alefacept did not show effectiveness."

Clinical • Review • Dermatology • Immunology • Vitiligo

Following over 12 months of interaction, two formal submissions and a Discussion Meeting with the EMA’s Scientific Advice Working Party (SAWP), the Committee for Medicinal Products for Human Use (CHMP), has issued Scientific Advice on CLINUVEL’s program evaluating SCENESSE as a systemic therapy for adults and adolescents with non-segmental vitiligo. (GlobeNewswire) - "The EMA proposed to evaluate the efficacy of SCENESSE in vitiligo based on a regulatory approach of 'totality of evidence', with T-VASI502 the primary endpoint while patient reported outcomes and clinical data from the other vitiligo studies with SCENESSE will be evaluated for final analyses of efficacy and safety...In particular, photographic evidence of change from baseline (CFB) will be used to assess primary and secondary endpoints, T-VASI50² and F-VASI75³, with an array of other related secondary objectives. In total, five patient and physician surveys are integrated in the study to record Patient Reported Outcomes...CUV107 to compare SCENESSE with adjunct NB-UVB vs NB-UVB monotherapy; CUV107 study (n=300) to commence in 2H 2026"

CHMP • New P3 trial • Vitiligo

Heterochiral co-assembly of β-strands and hairpins affords stereocomplexed peptide hydrogels for drug delivery. (PubMed, J Control Release) - "Utility of this material was demonstrated through the delivery of afamelanotide, a peptide melanocortin 1 receptor agonist. The stereocomplexed delivery system offers slow sustained release of active peptide drug over several months. The heterochiral material is designed to be versatile to allow the delivery of other classes of drug by their simple ligation to the β-strand component of the system."

Journal

New and currently investigated pharmacotherapies for the erythropoietic protoporphyrias: spotlight on dersimelagon and bitopertin. (PubMed, Expert Opin Pharmacother) - "However, safety and efficacy of dersimelagon and bitopertin need to be further characterized. Moreover, to ensure benefit for patients and access to therapy after regulatory approval, it also would be important to generate data on the relative safety and efficacy as compared to afamelanotide."

Journal • Review • Genetic Disorders • Metabolic Disorders • Pain • Rare Diseases

A Family With X-linked Protoporphyria (AAD 2026) - "Treatment was initiated with afamelanotide, an α-melanocyte-stimulating hormone analog, administered as a 16mg subcutaneous implant every 60 days to increase photoprotective eumelanin...Shortly after her diagnosis, her 11-year-old daughter was also diagnosed, highlighting the critical importance of screening at-risk family members. Early identification allows for timely intervention, genetic counseling, and proactive monitoring to manage the debilitating cutaneous symptoms and potentially severe hepatic complications of XLP."

Genetic Disorders • Hematological Disorders • Hepatology • Liver Failure • Metabolic Disorders

Afamelanotide improves quality of life and light tolerance in Austrian erythropoietic protoporphyria patients. (PubMed, J Dtsch Dermatol Ges) - "The real-world data of the Austrian cohort confirm the effectiveness and safety of afamelanotide in EPP patients."

HEOR • Journal • Genetic Disorders • Metabolic Disorders • Pain

Adjunctive use of Polypodium leucotomos extract in patients with erythropoietic protoporphyria: An exploratory study. (PubMed, Photochem Photobiol) - "Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) cause severe photosensitivity, resulting in significant quality of life (QoL) impairment. Adjunctive PLE improved short-term QoL in participants with incomplete symptom control on afamelanotide alone and was well tolerated. Larger studies are warranted."

Journal • Genetic Disorders • Metabolic Disorders

Congenital Erythropoietic Porphyria with Persistent Severe Biochemical Abnormalities and a Non-Mutilating Clinical Course: A Case Report. (PubMed, Reports (MDPI)) - "The patient experienced marked improvement in photosensitivity and burning pain after initiation of afamelanotide, without the need for transfusion therapy or stem cell transplantation. This case highlights the heterogeneity of CEP, the importance of long-term biochemical follow up, and the potential role of afamelanotide in improving quality of life for selected patients with CEP."

Journal • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Pain • Rare Diseases • Transplantation

Investigation of the stability profile of therapeutic α-MSH analogue: Insights from liquid chromatography-high resolution mass spectrometry analysis of afamelanotide. (PubMed, J Pharm Biomed Anal) - "To enable comprehensive characterization, the analysis was coupled with ultra-high-performance liquid chromatography-high resolution tandem mass spectrometry (UHPLC-HRMS/MS), where collision-induced dissociation yielded abundant and accurate fragmentation patterns, enabling the detailed structural elucidation of the products. While this work has identified several degradation pathways such as truncation, methylation, deacetylation, and oxidation, it also establishes complete stability profile of α-MSH analogue, thus offering key insights for the rational design of robust drug formulations."

Journal • Genetic Disorders • Metabolic Disorders

From darkness to light: Case report on afamelanotide-treatment in a 9-year-old child with erythropoietic protoporphyria. (PubMed, JAAD Case Rep) - No abstract available

Journal • Genetic Disorders • Metabolic Disorders • Pediatrics

Hormones and skin pigmentation: fundamentals and clinical relevance (PubMed, Dermatologie (Heidelb)) - "Two synthetic melanocortins, afamelanotide (NDP-α-MSH) and setmelanotide, are currently approved in Germany. Autoimmune thyroid disorders and diabetes are associated with non-segmental vitiligo. Melatonin appears to have a lightening effect on skin pigmentation by melanin."

Journal • Review • Dermatology • Dermatopathology • Diabetes • Endocrine Disorders • Immunology • Metabolic Disorders • Rare Diseases • Vitiligo • IGF1

New pharmacotherapies for the erythropoietic protoporphyrias: an analysis of trial protocols from a patient perspective. (PubMed, Orphanet J Rare Dis) - No abstract available

Journal • Genetic Disorders • Metabolic Disorders • Rare Diseases

Letter to the Editor - Qualitative evidence submitted by patients to NICE: need for more quality or unrealistic and unfair requirements? (PubMed, J Comp Eff Res) - No abstract available

Journal • NICE • Genetic Disorders • Metabolic Disorders • Rare Diseases

Burden of illness and unmet needs in patients with erythropoietic protoporphyria and X-linked protoporphyria: A large US nationwide claims analysis. (PubMed, J Manag Care Spec Pharm) - "Although only 7.6% of patients with EPP/XLP received treatment for EPP/XLP, most commonly afamelanotide (3.9%), most (68.4%) used medication related to EPP/XLP-associated comorbidities, including narcotics (46.3%), nonsteroidal anti-inflammatory drugs (38.2%), and antidepressants (35.1%). Patients with EPP/XLP experienced substantially higher HRU and costs compared with matched comparators, yet few received EPP/XLP-specific treatment. This highlights the need for new effective and accessible treatments that could improve patient outcomes and alleviate the broader disease burden."

Journal • Genetic Disorders • Metabolic Disorders • Pain

Polymorphism of Melanocortin Receptor Genes-Association with Inflammatory Traits and Diseases. (PubMed, Diseases) - "Among them, α-MSH analogs play a role in atopic dermatitis and scleroderma, and MC1R agonist Dersimelagon has shown effectiveness in systemic sclerosis. The FDA has recently approved the repository corticotropin injection (RCI) to treat sarcoidosis. The FDA has also approved various melanocortin agonists, i.e., Bremelanotide, Afamelanotide, and Setmelanotide, for the treatment of hypoactive sexual desire disorder, Erythropoietic protoporphyria, and obesity, due to pro-opiomelanocortin and leptin receptor deficiency, respectively. Therefore, this review aims to summarize the function and genetic polymorphism of melanocortin receptors, regulatory pathways involving MCRs, and the existing evidence of the prime effect of MCRs on inflammatory responses via different mechanisms and their potential therapeutic use in inflammatory diseases."

Journal • Review • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • Atopic Dermatitis • CNS Disorders • Depression • Dermatitis • Dermatology • Diabetes • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Immunology • Inflammation • Inflammatory Bowel Disease • Major Depressive Disorder • Melanoma • Metabolic Disorders • Multiple Sclerosis • Obesity • Ocular Inflammation • Oncology • Ophthalmology • POMC-null Obesity • Psychiatry • Reperfusion Injury • Respiratory Diseases • Rheumatology • Sarcoidosis • Scleroderma • Sexual Disorders • Solid Tumor • Systemic Sclerosis • Type 2 Diabetes Mellitus • Uveitis • LEP • LEPR

Pharmacokinetics and safety of afamelanotide in adolescent and adult erythropoietic protoporphyria (EPP) (EADV 2025) - "In conclusion, PK profiles for afamelanotide were higher in adolescents versus adults and consistent with previous data. Afamelanotide was well tolerated in both adolescents and adults. Data from this study adds to the safety and efficacy profile for afamelanotide in the adolescent population."

Clinical • PK/PD data • Genetic Disorders • Metabolic Disorders • Pediatrics

Afamelanotide: A Novel Promising Treatment for Vitiligo Case Studies from the Randomized CUV105 Clinical Trial (EADV 2025) - "Afamelanotide is a melanogenesis-stimulating agent able to accelerate the repigmentation process in vitiligo, and promote more rapid and sustained repigmentation when used in combination with NB-UVB phototherapy. Character count (excl headings and spaces): 2,346"

Case study • Clinical • CNS Disorders • Dermatology • Immunology • Vitiligo

A systematic review of case series and clinical trials investigating systemic oral or injectable therapies for the treatment of vitiligo (EADV 2025) - "Systemic treatments for vitiligo have been successful in controlling lesions without notable side effects. OMP, Methotrexate, Azathioprine, Cyclosporine, Mycophenolate mofetil, Simvastatin, Apremilast, Minocycline, Afamelanotide, Tofacitinib, Baricitinib, Antioxidants, and oral/injectable corticosteroids are effective treatment methods. However, oral zinc and alefacept did not show effectiveness."

Clinical • Review • Dermatology • Immunology • Vitiligo

Erythropoietic protoporphyria in childhood: clinical clues, missed diagnoses and emerging therapy. (PubMed, Eur J Pediatr) - "It also reviews emerging treatment options such as afamelanotide, already approved for adults, with promising adolescent data. These advances must now be widely communicated and translated into equitable access for children across Europe."

Journal • Review • Dermatology • Genetic Disorders • Hematological Disorders • Hepatology • Metabolic Disorders • Pain • Pediatrics

Silica-Assisted Solid-Phase Peptide Synthesis (SiPPS). (PubMed, J Pept Sci) - "The strategy was well demonstrated for the synthesis of H-YSSFL-NH2, linear oxytocin, angiotensin II, and afamelanotide using a silica-based support (Fmoc-Rink amide SiliaBond manufactured from SiliCycle Inc.). The peptides were found to have more than acceptable purity, although there was a loss in overall yields."

Journal

Afamelanotide in Managing Cutaneous Phototoxicity in Erythropoietic Protoporphyria: A Scottish Perspective. (PubMed, Clin Exp Dermatol) - No abstract available

Journal • Genetic Disorders • Metabolic Disorders