Into the Light: Afamelanotide and the Treatment of Erythropoietic Protoporphyria in the United States.

Abstract

BACKGROUND:

Erythropoietic protoporphyria (EPP) is a rare disease that causes disabling cutaneous photosensitivity with pain and burning sensations. In 2019, afamelanotide, an α-melanocyte-stimulating hormone analogue, was approved in the United States for treatment of EPP. In this study, patients receiving afamelanotide filled out questionnaires assessing the benefit of treatment. Outcomes measured included: return to normal activities, experience of phototoxic reactions, effect on patient confidence, and more. Patients ranked their experience on a descriptive scale ranging from "very much" to "never".

RESULTS:

Prior to treatment, 75% of patients indicated that EPP affected their lives "very much" or "a lot". This number fell to 11% after the 1st implant and to 0% after each subsequent implant. The number of patients that willingly ventured outside increased with each subsequent implant.

CONCLUSION:

The results of this study clearly show that afamelanotide treatment can dramatically and positively impact the lives of EPP patients.  Citation: Resnik SR, Targett D, Resnik BI. Into the light: afamelanotide and the treatment of erythropoietic protoporphyria in the United States. J Drugs Dermatol. 2023;22(9):941-949. doi:10.36849/JDD.7126R1.

• Authors: Sydney R Resnik, Darren Targett, Barry I Resnik
• Journal: Journal of drugs in dermatology : JDD (J Drugs Dermatol) Vol. 22 Issue 9 Pg. 941-949 (Sep 01 2023) ISSN: 1545-9616 [Print] United States
• PMID: 37683058 (Publication Type: Journal Article)

Chemical References

• afamelanotide
• alpha-MSH

Topics

• Humans
• alpha-MSH (adverse effects)
• Protoporphyria, Erythropoietic (diagnosis, drug therapy)
• Dermatitis, Phototoxic
• Pain