According to CheckOrphan, Clinuvel Pharmaceuticals recently announced that their drug Scenesse has received a Prescription Drug User Fee Act (PDUFA) date from the FDA, set for July 8th of this year. This is the date by which the FDA must be done reviewing the drug. Clinuvel submitted a New Drug Application (NDA) for Scenesse, which aims to treat erythrohepatic protoporphyria (or EPP), back in June of 2018.

About Erythropoietic Protoporphyria

Erythropoietic Protoporphyria is a kind of porphyria, which is a group of genetic disorders characterized by problems with the production of heme. Heme is an important chemical group in hemoglobin molecules, which are responsible for transporting oxygen throughout the body.

The condition is caused by mutations in the FECH gene, which is responsible for coding the production of an enzyme called ferrochelatase. Ferrochelatase is involved in the production of the body’s heme. This defective FECH gene can limit the activity of ferrocheletase, leading to the accumulation of protoporphyrin (essentially heme) in the blood, bone marrow, and skin. This buildup can cause liver damage, gall stones, and almost universally, an extreme sensitivity to UV light sources.

Presently, no purpose-built treatments for EPP patients have been approved in the United States, though Clinuvel hopes to change that in the coming months.

Scenesse Awaits a Ruling

By July 8th the FDA must have issued a ruling on Scenesse’s New Drug Application. In 2008 the FDA granted Scenesse orphan drug designation, bestowing a slew of perquisites on Clinuvel including tax incentives, research subsidies, and most famously, a seven-year period of marketing exclusivity pending the final approval of Scenesse’s NDA.

Scenesse is an injectable inplant containing afamelanotide, a synthetic peptide that researchers hope can help control phototoxic and anaphylactic reactions in people with EPP by mimicking an α-melanocyte stimulating hormone. Melanocytes are cells that produce melanin, a dark pigment found in the hair and skin that’s responsible for the tanning effect from sunlight.

Clinuvel has conducted five clinical trials for Scenesse in EPP patients, including two that were conducted here in the United States. Patients in the studies did seem to enjoy a noteworthy increase in the amount of time they could spend outside without risking phototoxicity.

Scenesse has already been approved in the European Union, where some experts are already prescribing it to patients with EPP. By this summer, Americans living with the condition will have their answer as to whether or not Scenesse will be available to them – hopefully early enough for them to catch some beach time.

Do you or does someone you know live with erythrohepatic protoporphyria? How much does it impact your daily routine? Would you be excited at the prospect of being able to spend more time in the sun? Share your thoughts with Patient Worthy!