Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients

• Afamelanotide 16mg implant (Drug), also known as SCENESSE®: afamelanotide 16mg implant will be administered to each patient during the study.

Primary outcomes

• Cmax (Maximum Plasma Concentration) [Baseline to Day 7]: Blood samples will be collected for analysis of afamelanotide plasma concentrations and understand what the maximum concentration in plasma is.
• AUC(0-t) (Area Under the Curve From Administration to Last Observed Concentration at Time t) [Baseline to Day 7]: Blood samples will be collected for analysis of afamelanotide plasma concentrations.

Secondary outcomes

• AUC(0-∞) (Area Under the Curve Extrapolated to Infinite Time) [Baseline to Day 7]

Sex: All, ages: 12 Years to 70 Years

Inclusion Criteria:

* EPP patients aged between 12 and 70 years
* BMI between 15 and 30 kg/m2
* >50 kg

Exclusion Criteria:

* Any personal or direct family history of melanoma
* Any significant history of allergy and/or sensitivity to any of the contents of study drug product or lignocaine or other local anaesthetics if used
* Any significant illness during the four weeks before the study screening period
* Any evidence of hepatic or renal impairment

Participant flow

Outcome results

• Cmax (Maximum Plasma Concentration) (Primary), Geometric mean, ng/mL: Afamelanotide 16 mg Implant 2.361 (1.85 to 3.013)
• AUC(0-t) (Area Under the Curve From Administration to Last Observed Concentration at Time t) (Primary), Geometric mean, h*ng/mL: Afamelanotide 16 mg Implant 95.415 (77.075 to 118.119)
• AUC(0-∞) (Area Under the Curve Extrapolated to Infinite Time) (Secondary), Geometric mean, h*ng/mL: Afamelanotide 16 mg Implant 114.406 (96.631 to 135.45)

Adverse events

Time frame: All adverse events were monitored from baseline until 3 months after treatment (approximately 3 months)

Other adverse events by term

• Study Protocol and Statistical Analysis Plan (PDF, 2024-02-23)