What is the basis of your opinion for quoting $10-15k?
I'll stick with projections based on comparisons as that is a sound valuation methodology. In that regard I have included some which were used to substantiate the BP $100k below.
FXS and Retts are diseases which have terrible serious side effects so there is no issue with reimbursement via welfare i.e. the parents are not "forking" out the $100-200k annually that is covered in part by the millions who do not suffer Retts and FXS. In short, that is social welfare.
Anyway, I am agreeable with lilypily, Tony and co. in that BP are being conservative and discounting the $$'s NEU could charge by a magnitude of circa 50% when quoting $100k. As I said I base this on actual comps in the orphan drug space and here are a few examples of existing drugs in the orphan space:
Cerezyme –Sanofi costs US$300k per patient and eliminates fatty clumps that damage the spleen,liver, lungs and bone marrow
Elaprase –Shire Plc –costs US$375k per year and treats hunter syndrome that causes flattened noses and breathing and brain damages and is only 2k patient market.
Cinryze –Viopharma costs US$417k per year and prevents hereditary angioedema which is swelling of the abdomen and lips
Kalydeco –Vertex costs US$294k per year and targets a genetic defect at the heart of lung disease and cystic fibrosis
I think you'll find that they're compelling enough and relative to these the $100k quoted should be considered fair and reasonable.
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