The set date for the FDA decision regarding approval of Scenesse is 10 days away.
The APF has held multiple small meetings, a Scientific Workshop attended by +125 patients and caregivers, and most recently, a Rare Disease Listening Session with the FDA. We have sent stacks and stacks of your letters and stories, mailed letters to key leaders at the FDA, sent photos of people with EPP both in an attack and burdened by protective clothing, shared regular updates of patient situations and media stories, and worked to characterize the true burden of this disease on a patient.
There are over 7,000 known rare diseases - only 5% of them have available treatment! The FDA has this opportunity to make Scenesse, for the treatment of EPP, one of them.