Ultragenyx Biopharmaceuticals, Inc. (RARE) Stock | Approved MAA filing


Ultragenyx Biopharmaceuticals, Inc.  (NASDAQ: RARE)


Shares of RARE have been veering upward once more following the announcement of a successfully filed Marketing Authorization Application (MAA) with the European Medicines Agency (EMA). Ultragenyx Biopharmaceuticals (RARE) submitted its application for the product Ace-ER (UX001), aimed at treating people with GNE Myopathy. If the European Commission gives their conditional approval in 2016 we can expect the stock to hurtle upwards once more. As with most biotech stocks, expect a rollercoaster ride until then and remember it can feel like a long way to the top.


RARE Overview


Ultragenyx Biopharmaceuticals is a company that produces products aimed at treating rare and ultra-rare diseases, for which there are currently no approved therapies. The company has seen its share of highs and lows this year. Starting the year at 43.88, the stock has since been oscillating between a value of roughly 80-130, since June, barely ever dropping below this, despite the fact that Morgan Stanley downgraded the rating to equal-weight in July. After its announcement that it had filed to the MAA, the stock rallied at 107.52 but shot back down a few days later. Should approval be obtained in 2016 for ACE-er from the European Commission, Ultragenyx will then apply with the FDA in 2017. What ensues is contingent upon whether the European Commission grants their approval for ACE-er to move into phase 3 trials.



Ultragenyx Biopharmaceuticals frequently appears on various lists of top biotech stocks to watch out for. The company has gained popularity due to various reasons. One of which is that Ultragenyx possess a broad pipeline of products aimed at the treatment of rare and ultra-rare diseases. Although constituting a fairly high risk investment, investors have seen high rewards since the company went public. Morgan Stanley however, downgraded the stock in July stating that the stock valuation is “stretch” and that there is higher execution risk on the company’s pipeline. Nevertheless, in the last two years the share has gone up 125.49%. Furthermore, not only have Ultragenyx successfully filed medication for patients with GNE Myopathy but they also released positive interim data from their Phase 2 study of the treatment of X-linked hypophosphatemia, a dominant form of rickets. Ace-ER maintains Orphan Drug designation in the U.S. and EU for HIBM. Apart from within Japan and other Asian territories, Ultragenyx has exclusive rights to the compound.


Company Profile

Ultragenyx, located in California, is a clinical-stage biopharmaceutical firm. It brings to market innovative products for the treatment of rare and ultra-rare diseases, focusing on serious, debilitating genetic diseases. Ultragenyx was founded in 2010. Since then it has already filed one product, ACE-er, with the MAA which if met with approval will be flooding the European markets and will also be submitted to the FDA in 2017. The company has a wide pipeline of potential products aimed at addressing diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.