DAY AT THE FDA with Mike Kenworthy,Ph.D and Matt Johnson
I was part of the Afamelanotide trial last summer and was fortunate enough to have received the real thing. Unless you have EPP, I cannot explain what a miracle that was. But I did get the chance recently to do just that at the FDA Office of Orphan Diseases. Another fellow EPPer, Matt Johnson, joined me. In my 62 years I had never met anyone with EPP outside of my family (I have two cousins with EPP). That was an experience in itself. Those of us with EPP know that we can never explain to someone without EPP what it is like, but listening to Matt Johnson was like reading my own thoughts. Matt and I spoke a couple of times during telephone conferences before we actually met at the FDA in Silver Spring, Maryland. Although our session was scheduled for 10:30am, we were asked to come early to meet with Dr.Timothy Cote, the head of the Office of Orphan Diseases. I arrived just before 9am and Matt was close behind. Dr. Cote had just returned from Israel two day prior and was sick the day before, so he was not in good spirits, but after a few minutes listening to Matt and me, he was beaming. He told us that his staff, like all bureaucratd, tends to lose site of their mission and was delighted that we were there to remind them that we are out there.
Dr. Bob Desnick, a member of the APF Advisory Board, was also to attend, but his flight from New York delayed him an hour. By the time we were ushered into the conference room over 30 doctors and health professionals were waiting to hear what we had to say. For an hour Matt and I regaled them with the inexplicable world of EPP and the miracle of Afamelanotide during which time we were videotaped. We tried to tell them of the unrelenting pain, the isolation and self-doubt, and ultimate despair. Then we told them about our experience Last Summer and how it had changed our lives. We weren't supposed to directly discuss this, but it just couldn't stay untold - so we did. Dr. Desnick translated what we had to say into meaningful medical speak, but I believe that our words had more impact.
Like most families, we have gone to the beach almost every summer, and every summer after 15 minutes, I am banished to the house and sit with my feet in a lobster pot of ice water for the next 5 days ?sipping? copious amounts of adult beverages. Needless to say this is hardly endearing to my wife and makes everyone wonder. But last summer, I spent hours on the beach with virtually no pain ? a certified miracle if ever there was one. Like most EPPers, I have never told anyone outside of my immediate family about my condition. My colleagues at the office began referring to me as Mr. Tan without ever knowing why.
But the most compelling thing that day at FDA was a video that Matt had brought. It was a video of the episode that led to his diagnosis. He was 11 at the time and had gone skiing with his family. The video showed the progress of the effect of EPP over the period of about 10 days. As most of EPPers know, even though the pain has past, we are not a pretty site after 10 days. It had a profound effect on our audience.
Drs. Cote and Mueller thanked us for our time and promised that they would advocate our case to the extent they could, but could promise nothing. They explained that there is concern that the drug would be used for ?off label? usage and that it would take time and effort to weigh the risks and benefits of a drug such as Afamelanotide. Dr. Mueller told us that she would share the video of our session with the FDA Center for Drug Evaluation and Research. I believe that this was a first and significant step in getting approval for the use of Afamelanotide, but I am not sure that the end is in sight. I live 45 minutes from the FDA campus and with the help of APF will continue to do whatever it takes to gain approval for use of Afamelanotide. Posted by American Porphyria Foundation at 10:17 AM
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