EPP
Erythropoietic protoporphyria (EPP) is a rare disease that causes extreme light sensitivity. This sensitivity is so extreme that being outside is extremely difficult for patients and many only come out of their homes at night.
The condition is a result of a defect in heme biosynthesis. It causes PPIX to accumulate in the blood and tissues of the body. Visible light photo-activates PPIX causing both extreme pain and damage to the tissue.
Until now, there have been no approved treatments for the disease in the United States.
Thankfully a new medication called Scenesse has just been approved by the FDA for the treatment of EPP.
Scenesse
What exactly is Scenesse? It’s an implant that goes underneath the skin.
Scenesse is Clinuvel’s only approved medication so far.
Within the next year it will be distributed directly to hospitals inn the United States.
The exchange of information between the EMA and the FDA is accelerating. This exchange will help medications like Scenesse become available to additional patient populations faster. Because they believe this medication will be beneficial for additional implications, the Clinuvel team knows that their work has just begun. Their goal is to “execute against due dates while containing the costs.”
About The Cost
Something incredible is that the company Clinuvel plans to price the drug at the same cost in every country. The concept of having a single price-point is very uncommon in the field of healthcare. But this way no one patient will be paying more for their medication in any one country.
The Timeline
- 2006: Clinical trials began in Europe
- 2008: Scenesse was given Orphan Drug Designation by the FDA
- 2010: Phase 2 trial was initiated in the US
- 2013: Phase 3 trial was initiated and completed in the US
- 2014: Scenesse was approved in Europe for adults
- 2016: FDA began reviewing data from all clinical trials and held a workshop for patients and their families to share their experiences with EPP
- 2017: Scenesse received Fast Track Designation by the FDA
- 2019: Scenesse was given Priority Review by the FDA
- 2019: Scenesse was approved for EPP by the FDA
You can read more about this new approval here.
