When you think of the Mediterranean, you probably picture sun-kissed white sand beaches, azure blue waters, and the exotic coastlines of Italy, Greece, and Morocco.What you probably don’t picture is recurrent, painful inflammation in the abdomen, chest, or joint, along with a fever, rash, or headache. You definitely don’t picture inflammation of the heart or the membranes of the brain and spinal cord, or, most painfully for gents, the testicles.
If you don’t picture these symptoms, then you’ve never heard of familial Mediterranean fever (FMF). According to the NIH, it typically occurs in people with Mediterranean ancestry (natch), especially people of Armenian, Arab, Turkish, or Jewish descent.
Only 1 in 200 to 1 in 1000 people in these populations is affected, so it’s an extremely rare condition. Episodes typically begin in infancy and childhood (but can begin much later); the severity of illness can vary, and so can the length of time between episodes.
There is no cure for FMF, and left untreated patients can develop amyloidosis from the build-up of abnormal proteins in organs and joints. To date there has been only one FDA-approved treatment, colchicine, for reducing inflammation.
But that may be changing soon. In May the FDA granted three new Breakthrough Therapy Designations for Ilaris® (canakinumab), a Novartis drug that’s used to treat some rare autoinflammatory diseases.
Breakthrough Therapy Designation basically means that a condition is so rare—and the treatment options are slim to non-existent—that if a promising treatment comes along, the FDA will work to expedite the review and approval process. Based on the results of a Phase III trial, the FDA will be working with Novartis to consider approving Ilaris for three indications, among them FMF.
While this is by no means a done deal—the FDA still has to review the information filed by Novartis and consider if the results seen in the trial merit approval—but the possibility of a new treatment option is awesome news for patients who haven’t had any luck controlling their symptoms with colchicine. Until the FDA hands down their decision, cross your fingers, close your eyes, and go to your happy place.Already ahead of you.