If you’re one of the very few people in U.S. living with familial chylomicronemia syndrome (FCS), the odds are good you that you know more about it than your family doctor.
Speaking of odds, can you believe only 1 out of 1 million people are diagnosed with FCS?!
FCS is an ultra-rare, hereditary lipid condition that causes disruption of enzyme activity, leading to high concentrations of triglycerides and chylomicrons in the blood. The result? Similar to other lipid affected conditions like FH, FCS can cause pancreatitis, skin lesions called eruptive xanthomas, abdominal pain, and more. And until recently, treatment options were nonexistent. But that’s all changing…
As reported by the official Nasdaq website Akcea Therapeutics, a subsidiary of Isis Pharmaceuticals, recently announced that its new FCS treatment volanesorsen has been granted orphan drug designation by the FDA.
Orphan Drug designation is for drugs serving a patient population <200,000—with an estimated one to two people out of a million living with FCS around the world, it definitely qualifies.
Did you know that only 10% of rare disease have approved orphan drugs? This is a huge win for familial chylomicronemia syndrome (FCS).
Volanesorsen is undergoing a phase III clinical trial right now, and Isis Pharma is working to begin another phase III trial for another indication later in 2015. The orphan drug designation also makes the company eligible for tax credits and other exemptions to help offset clinical trial expenses. All of which means that once volanesorsen is approved, both FCS patients and Isis Pharma will have a lot to celebrate!
We are a heavily drugged society (over-the-counter people) that sometimes glances over the new drugs being approved daily. This is Different! If you are an advocate for rare lipid conditions like FCS or FH, or any rare disease for that matter, share this article and spread hope for the few but important FCS patients out there.