We issued an updated research report on BioMarin Pharmaceutical Inc.BMRN on Jan 25.
BioMarin should continue to see growth in both its orphan disease drugs - Vimizim and Kuvan - in 2017. Vimizim sales continue to be driven by robust patient growth and penetration in additional markets. The product should perform well with existing and new patient referrals converting to commercial therapy. Products like Kuvan are also performing well and its North American sales continue to propel on the back of increase in new patients and high levels of adherence. Internationally, uptake has been solid and the acquisition of the drug's global rights has opened a stream of orders directly from the majority of the top markets to the company. BioMarin continues to see growth in both Vimizim and Kuvan in 2017.
The company has a robust pipeline with several data readouts lined up in the coming quarters. The candidates include pegvaliase (PKU, regulatory application in the U.S. planned for the second quarter of 2017) and vosoritide (achondroplasia,). Also, BioMarin has several early-stage and mid-stage candidates in its pipeline like Brineura (treatment of children with CLN2 disease, response from the FDA is expected by Apr 27, 2017 and a decision in the EU anticipated in the third quarter of 2017), BMN 270 (hemophilia A) and NAGLU (Sanfilippo B syndrome or MPS IIIB).
Successful development and commercialization of these candidates in its pipeline will help drive long-term growth at BioMarin.
However, BioMarin is facing generic threat for Kuvan. The company acquired all global rights to Kuvan, excluding Japan and pegvaliase from Merck Serono-the biopharma business of Merck KGaA MKGAF , in Jan 2016. Kuvan is one of the most promising drugs at BioMarin and has been performing well since its launch. As a result, the earlier-than-expected entry of generics would be a huge blow for the company. In Feb 2016, BioMarin filed a patent infringement lawsuit in the U.S. District Court for the District of New Jersey against Endo International plc's ENDP Par Pharmaceutical for Kuvan.
BioMarin has suffered quite a few regulatory setbacks related to its pipeline candidates, Brineura and Kyndrisa. Delayed approvals or development setbacks could have a negative impact on the stock. Also, the company's top line is under pressure due to political and macroeconomic instability in Brazil and the Middle East. However, BioMarin expects the situation to improve in 2017 with break-even or better results.
In Jan 2015, BioMarin acquired a Dutch biotech company Prosensa, in an all-cash transaction valued at $751.5 million, to boost its rare disease pipeline. The acquisition was assumed to be a strategic move. The acquisition added a late-stage Duchenne muscular dystrophy (DMD) candidate Kyndrisa, which was considered to be a synergistic fit for BioMarin's rare disease pipeline. Nevertheless, BioMarin failed to clear regulatory hurdles related to Kyndrisa and ultimately decided to drop development of the candidate. However, biopharmaceutical company Sarepta Therapeutics, Inc. SRPT , ceived a boost with the FDA approval of Exondys 51 for DMD. The FDA approval makes Exondys 51 the first DMD treatment to gain approval in the U.S. Also, a marketing application is under review in the EU.
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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.