Isis Says Ultra-Orphan Use Promises Speedier Approval For ISIS-APOCIIIRx
This article was originally published in The Pink Sheet Daily
Sharing data for just three patients with the ultra-rare genetic disease familial chylomicronemia, Isis says starting with this indication will allow market entry one year earlier.
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Genzyme and Isis are playing up mipomersen’s intrinsic qualities as they prepare to go head-to-head against Aegerion’s Juxtapid in the small homozygous familial hypercholesterolemia market. Kynamro also will carry a price tag that is $119,000 less than Juxtapid’s.
Part 2: High costs are built into the rare disease business model. Participants at a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference talked about how much they are feeling the pressure on pricing.
Part 1: Execs from rare disease-focused companies weighed in on investor sentiment during a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference.