MT-1, Melanotan I (afamelanotide)

In these two studies, 168 patients with erythropoietic protoporphyria (EPP), a rare condition with strong, painful light reactions in the skin, received either an afamelanotide implant under the skin or placebo. Those who received afamelanotide could spend longer in direct sunlight without pain, and in the European study there were fewer light-induced reactions. Quality of life improved in both studies. Side effects were mostly mild. The authors conclude that there was longer pain-free sun exposure and better quality of life in EPP.

In this long-term study, 115 EPP patients were followed for up to 8 years while receiving a total of 1,023 afamelanotide implants at European treatment centres. Most tolerated light better, and quality of life rose from around 31% to about 74% of the maximum score and stayed there. Only three patients felt the treatment did not meet their expectations, while some stopped for other reasons such as pregnancy or finances. Side effects were mild, mostly nausea. The authors judge efficacy and safety to be good.

In this European observational study, 39 EPP patients were followed for three years during treatment with afamelanotide. The time they could spend in sunlight without painful skin reactions increased from about 10 minutes to around 180 minutes. Pain during the worst reactions decreased, and quality of life was high (just over 81%). Almost all followed the treatment as recommended. The authors consider afamelanotide highly effective under real-world conditions, and propose sun tolerance time as a useful measure in further studies.

In this American study, 29 patients with protoporphyria (EPP or X-linked protoporphyria) were followed while receiving afamelanotide in 2021–2022. Among those who received at least two implants, time in sunlight before symptoms increased from about 12.5 to 120 minutes, and quality of life improved. However, the researchers found no improvement in protoporphyrin levels in the blood or in liver values. The study thus shows clear clinical benefit for light tolerance and quality of life, but no change in these laboratory values.

In this study, 65 fair-skinned participants received injections of a potent, synthetic variant of melanocyte-stimulating hormone over three months. The skin's content of the brown pigment melanin increased considerably in everyone treated, most in those with the fairest skin at baseline. After UV irradiation, the number of sun-damaged skin cells was more than halved, and the formation of certain UV lesions in the DNA (thymine dimers) was reduced by 59%. The study suggests that increased melanin may give fair-skinned people some protection against sun damage.

In this study of 55 people with widespread vitiligo (white patches in the skin), narrowband UV-B light therapy was compared with the same light therapy combined with an afamelanotide implant. The combination gave better and faster repigmentation of the skin than light therapy alone, particularly on the face and arms, and most clearly in people with darker skin types. Side effects included skin redness, as well as nausea and minor infections in the combination group. The authors conclude that the combination gave a clearly better result.

Based on the title: This study looked at afamelanotide implants combined with narrowband UV-B light therapy for the treatment of non-segmental vitiligo in people of Asian background.

In this small study, 5 patients with solar urticaria (hives triggered by sunlight) received one afamelanotide implant in winter. The skin's melanin level increased within a week and remained raised. On light testing, the skin reactions (weals) became smaller across a broad range of wavelengths, and the tolerance threshold for light increased. Because of the very low number of participants the findings are uncertain, and the authors consider that more research is needed under ordinary summer conditions.

This review article examines how well afamelanotide works and is tolerated in erythropoietic protoporphyria (EPP). Afamelanotide is a synthetic hormone given as an implant under the skin. In the phase 3 study CUV039 patients tolerated light better: they could spend longer in direct sunlight without pain, and it took longer before the first signs of a light reaction appeared. The treatment was generally well tolerated, with headache and reactions at the implant site as the most common side effects.

This review article discusses the use of afamelanotide in protoporphyria, a disease with severe intolerance of sunlight. It describes the disease's characteristics, inheritance and existing treatment, as well as how melanocyte-stimulating hormone works and why afamelanotide is used. The authors describe the results from several phase 2 and 3 studies as significant, but the effect was not very large in absolute terms. The safety profile is considered favourable. They hope for future dosage forms suited to children, who are hardest hit.