
Faced with slack pipelines and increasing payor demands, big pharma CEOs spouted the words "niche" and "orphan" more often then "primary care" and "blockbuster" this year (though they have been biting their tongues behind the scenes or crossing their fingers behind their back).
Partnering with Amicus required only a small upfront on the part of GSK, which paid $60 million for a 19.9% equity stake in Amicus in addition to worldwide rights to Amigal. GSK also agreed to pay $170 million in milestones, clinical development costs and tiered double-digit royalties on sales. But it stands to gain in return. As a reference point, the market-leading enzyme replacement therapy for Fabry disease – Genzyme's Fabrazyme – generated $494 million in sales in 2008, the year before Genzyme's manufacturing problems negatively impacted sales. GSK is betting there is plenty of upside potential for a more convenient oral brand. And with Genzyme embroiled in a manufacturing debacle and a hostile takeover battle by Sanofi-Aventis, it opens the door for rivals to stake a claim for the space.
For Amicus, the deal provides enough cash to see it through to the US approval of Amigal, according to management, and getting GSK on board as a partner helps validate its pharmacological chaperone technology.
Whether or not big pharmas' increasing emphasis on rare diseases ultimately plays out in its favor is yet to be seen, but we think giving it a serious shot – at least as one platform in a multi-pronged growth strategy – is worth a DOTY nod.
-- JESSICA MERRILL
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.