"I'm very skeptical that this drug will get past this niche population."
It's all in how a "niche" is defined. As far as I can see (and anyone can correct me if I'm wrong) the MOA for mipomersen is at the LDL production mRNA level regardless of the cause of the excessive production of LDL. There are plenty of heterogenous familial hypercholesterolemia patients as well as idiopathic hypercholesterolemia patients who would fit this niche product.
If there are 600,000 patients with some form of familial hypercholesterolemia, I would define that as a pretty large niche.
ISIS has one antisense product out, Vitravene for cytomegalovirus retinitis (hits AIDS patients frequently). The cost of each vial for treatment is about $750 as best as I can find. Similar pricing for mipomersen brings a 10% penetration for monthly injections for those 600,000 patients to $540 million in sales.
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