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BioMarin Pharmaceutical Inc. (BMRN - Analyst Report) announced that it has completed dosing the first patient in a phase I/II study (n~22) on its pipeline candidate BMN 190. The study is designed to evaluate patients suffering from neuronal ceroid lipofuscinosis type II (NCL-II), a form of Batten disease.

The primary objective of the 48 week open-label, dose-escalation study is to assess the tolerability, safety and efficacy of the candidate. The effectiveness in treating the disease will be evaluated by using an NCL-II-specific rating scale score in comparison with the historical data following 48 weeks of BMN190 therapy. The secondary key points of the study are to assess the impact of BMN190 therapy on brain atrophy compared with the historical data following 48 weeks treatment. The study also intends to characterize pharmacokinetics as well as immunogenicity.  

The news on BMN 190 comes close on the heels of the research and development (R&D) day at BioMarin where the company provided an update on its pipeline. The most important pipeline candidate 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 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.

BioMarin also threw light on the development of early-stage candidates in its pipeline. 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), Biogen Idec Inc. (BIIB - Analyst Report) and Actelion Ltd. (ALIOF) are well placed with a Zacks Rank #1 (Strong Buy).

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