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BioMarin Pharmaceutical Inc. (BMRN - Analyst Report) threw light on its pipeline candidates at its research and development day on Sep 17, 2013. The most important pipeline at BioMarin is Vimizim. The candidate is being developed for the treatment of patients suffering from mucopolysaccharidosis Type IVA (MPS IVA) or morquio A syndrome.

The candidate is under review in the U.S. and the EU. BioMarin is also seeking approval of the candidate in Brazil. The target date in the U.S. is Feb 28, 2014. BioMarin stated in its press release that prior to the final decision an advisory panel of the U.S. Food and Drug Administration (FDA) will review Vimizim’s marketing application on Nov 19, 2013. The European approval of Vimizim is also on track with the European Medicines Agency (EMA) validating Vimizim marketing authorization application and granting it accelerated review status. Consequently, BioMarin expects the EMA’s Committee for Medicinal Products for Human Use to render an opinion on the marketing application before Dec 31, 2013.

Another interesting candidate in BioMarin’s pipeline is PEG-PAL, which is currently in phase III development for the treatment of phenylketonuria (PKU). Data from the study is expected by the end of next year.

The pipeline at BioMarin also includes BMN-673 for genetically defined cancers. The company expects to initiate a phase III study on the candidate in deleterious germline BRCA mutation metastatic breast cancer shortly.

Another pipeline candidate is BMN-701, being developed to combat Pompe's disease. In Mar 2013, the company announced positive results on BMN-701. BioMarin announced in its press release that the FDA has accepted Maximal Inspiratory Pressure as the primary endpoint of the proposed phase II/III switching study on the candidate in patients suffering from late-onset Pompe’s disease who have been previously treated with Sanofi’s  (SNY - Analyst Report) Myozyme/Lumizyme (alglucosidase alfa). BioMarin also threw light on the ignition of the study in Europe. The company is currently in discussions with European regulatory authorities on the study. Following the discussions, the study on BMN-701 is expected to be initiated by year end/Mar 31, 2014.

BioMarin announced encouraging news on BMN 111 in children suffering from achondroplasia. The FDA lifted the clinical hold on the candidate, following submission of modified data and withdrawal of the disputed phase II protocol. The regulatory authorities in the U.S. as well as in Europe have agreed that a phase II study for the indication can be initiated without additional data. BioMarin intends to commence its first global study in pediatrics with achondroplasia by year end/first quarter of 2014.

BioMarin also stated that it has initiated a 48 week phase I/II study (n~22) on BMN 190 for treating patients suffering from Batten disease. The successful development and commercialization of the robust pipeline will help drive long-term growth at BioMarin, which currently has four marketed products.

BioMarin currently carries a Zacks Rank #3 (Hold). Companies like Gilead Sciences Inc. (GILD - Analyst Report) and Actelion Ltd. (ALIOF) are well placed with a Zacks Rank #1 (Strong Buy).

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