Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Melanocortin 1 receptor agonists

7 evidence topics · 8 sources

Approved indication and administration of afamelanotide

“SCENESSE is a melanocortin 1 receptor (MC1-R) agonist indicated to increase pain free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP). (1)” (opens the source at this quote in a new tab)
“SCENESSE should be administered by a healthcare professional who is proficient in the subcutaneous implantation procedure and has completed training prior to administration. (2.1)” (opens the source at this quote in a new tab)
“A single SCENESSE implant is inserted subcutaneously above the anterior supra-iliac crest every 2 months.” (opens the source at this quote in a new tab)
“The safety and effectiveness of SCENESSE have not been established in pediatric patients.” (opens the source at this quote in a new tab)
“The most common adverse reactions (incidence > 2%) are implant site reaction, nausea, oropharyngeal pain, cough, fatigue, dizziness, skin hyperpigmentation, somnolence, melanocytic nevus, respiratory tract infection, non-acute porphyria, and skin irritation (6).” (opens the source at this quote in a new tab)

European prescribing restrictions and authorisation basis for afamelanotide

“SCENESSE should only be prescribed by specialist physicians in recognised porphyria centres and administration should be performed by a physician trained and accredited by the marketing authorisation holder to administer the implant.” (opens the source at this quote in a new tab)
“The safety and efficacy of afamelanotide in children and adolescents aged to 17 years have not yet been established. No data are available.” (opens the source at this quote in a new tab)
“This medicinal product has been authorised under ‘exceptional circumstances’.” (opens the source at this quote in a new tab)

Randomized trial results for afamelanotide

“In the U.S. study, the duration of pain-free time after 6 months was longer in the afamelanotide group (median, 69.4 hours, vs. 40.8 hours in the placebo group; P=0.04). In the European Union study, the duration of pain-free time after 9 months was also longer in the afamelanotide group than in the placebo group (median, 6.0 hours vs. 0.8 hours; P=0.005), and the number of phototoxic reactions was lower in the the afamelanotide group (77 vs. 146, P=0.04).” (opens the source at this quote in a new tab)

Observational effectiveness of afamelanotide

“A total of 117 patients with EPP (59 women [50.4%]; mean [SD] age, 43.0 [15.5] years) were treated with afamelanotide. Nearly all patients continued treatment (115 [98%]) with a median (interquartile range) follow-up of 2.0 (1.3-2.1) years. Compared with baseline, mean time spent outside during treatment increased significantly by an added 6.1 hours per week (95% CI, 3.62-8.67; P < .001). Mean quality of life score improved significantly by 14.01% (95% CI, 4.53%-23.50%; P < .001). Phototoxic reactions were less painful (β, -0.85; 95% CI, -1.43 to -0.26; P < .001), but there was no significant difference in number or duration of reactions.” (opens the source at this quote in a new tab)

“The quality of life (QoL) scores, measured by an EPP-specific questionnaire, were 31 ± 24% of maximum prior to afamelanotide treatment, rose to 74% after starting afamelanotide and remained at this level during the entire observation period. Only minor adverse events attributable to afamelanotide, predominantly nausea, were recorded.” (opens the source at this quote in a new tab)

Guideline position on afamelanotide

“Therefore, afamelanotide is recommended for the prevention of phototoxic symptoms in EPP ( Table 2” (opens the source at this quote in a new tab)
“For patients using afamelanotide, total body skin examinations by a dermatologist are recommended every 6 months” (opens the source at this quote in a new tab)
“The drug is available in the US and Europe, however access is limited in the US due to the high cost and restricted sites for administration” (opens the source at this quote in a new tab)
“There is no data on the safety of afamelanotide during pregnancy so this cannot be recommended for pregnant women” (opens the source at this quote in a new tab)

Observed treatment duration and adherence in United States claims data

“Among the 27 patients with EPP/XLP who used afamelanotide during the follow-up period, the median duration of treatment was 5.68 months” (opens the source at this quote in a new tab)
“The mean PDC was 0.74 (SD = 0.23) at 6 months, 0.68 (SD = 0.21) at 12 months, and 0.63 (SD = 0.29) at 24 months, and only 39%-50% of patients had PDC greater than or equal to 0.8 in these periods” (opens the source at this quote in a new tab)

Cumulative global exposure to afamelanotide

“Over 18,500 SCENESSE® implants have been administered to EPP patients globally, demonstrating the sustained success and safety of the program, which commercially has achieved a compound annual growth rate (“CAGR”) of 35% since fiscal year 2016” (opens the source at this quote in a new tab)