News on the phase III results of BioMarin's orphan drug GALNS created a huge upward surge in shares of BMRN - 31% on November 5th.
GALNS treats an extremely rare condition known as Morquio A Syndrome (or MPS IVA) which only occurs in about 1,500 people in the United States. After meeting its primary endpoint, we can expect BioMarin to submit GALNS' NDA relatively soon. Due to the rarity of the disease and the lack of any treatment, I expect that the FDA will grant priority review to the GALNS NDA as well, which will shorten the review time from ten to six months. This means that we should expect a FDA decision some time in the middle of year 2013.
Although it seems that GALNS is a virtually done deal in terms of approval, BioMarin has gotten quite expensive after the news hit as described in a recent article I wrote. GALNS isn't really worth the $1.4 billion that the market added to BioMarin's market cap due to the extremely small number of Morquio A Syndrome patients, and looks a bit expensive.