Isis Says Ultra-Orphan Use Promises Speedier Approval For ISIS-APOCIIIRx
This article was originally published in Pharmaceutical Approvals Monthly
Executive Summary
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.
You may also be interested in...
Genzyme/Isis Build Simple, No Fuss Case For Weekly Cholesterol Injectable Kynamro
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.
Rare Disease Roundtable: Dealing With Pricing Pressure
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.
Rare Disease Roundtable: How Rare Is Too Rare?
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.