- US NDA and EU MAA have been accepted for review
- Expect FDA advisory committee meeting late 3q or early 4q 2012
- Expect US approval late 2012 and EU approval first half 2013
- Project profitability in 2014
- Working on patient registry for homozygous familial hypercholesterolemia (HoFH), won't say how many patients are on it until closer to launch
- Expect mipomersen and lomitapide to be revieweed at same panel. Topics of discussion could include trial design, liver safety, REMS
- AEGR has submitted a proposed REMS to FDA in NDA filing focused around strict on-label use and monitoring of liver enzymes.
- Don't see a place for PCSK9 drugs in HoFH
- Now can't forecast a date for access program/reimbursement in France (had been projecting by year-end 2011)
- Need to reformulate before starting pediatric trials -won't start until 2013, and probably after initial regulatory decisions. Need to get same bioavailability, stability, palatability for whatever presentation given to kids - so even if plan to open capsules and mix with food, would have to do the work. Liquid formulation has not been tried but should be technically feasible.
- Market size estimate ~3000 patients in US holds up across multiple methods of calculation so far.
- In these patients, neither mipomersen nor lomitapide is going to get all patient to goal LDL levels
- Intellectual property: Excited about patent issued in 2011 (administration patent through 2025-2026 in US/EU). Composition of matter (COM) patent expires in 2015, Hatch-Waxman extension through mid-2020. 10 years EU market exclusivity. Patent term extension for COM in US "yet to be determined"
Aegerion Pharma $AEGR held its first quarter 2012 earnings call on May 3rd. Here are a few quick notes regarding their drug candidate lomitapide and its potential competitor KYNAMRO (mipomersen) from Isis Pharma $ISIS.
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