BioMarin Pharmaceutical Inc. (BMRN) has a robust pipeline with several data readouts expected this year. 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 expected to be approved in the U.S. later in the year (target date: Feb 28, 2014) following the positive opinion of a panel of the U.S. Food and Drug Administration in Nov 2013. BioMarin is also seeking approval of the candidate in the EU. Approval of the candidate has also been sought in other countries like Canada.
Other interesting candidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently undergoing phase III development for the treatment of PKU. The pipeline at BioMarin also includes BMN-673 (a poly polymerase inhibitor). In Oct 2013, the company initiated a phase III study on BMN 673 in germline BRCA (gBRCA) breast cancer. Another pipeline candidate is BMN-701, which is being developed to combat Pompe's disease. Moreover, a 48 week phase I/II study (n~22) on BMN 190 for treating patients suffering from Batten disease is underway. BMN 111, for treating children suffering from achondroplasia, is another interesting pipeline candidate at BioMarin. The successful development and commercialization of the robust pipeline will help drive long-term growth at BioMarin.
In a move, aimed to strengthen its early stage pipeline, the biopharmaceutical company inked a deal with Repligen Corporation for pre-clinical compounds earlier in the week. Under the terms of the deal, BioMarin is making an upfront payment of $2 million to Repligen for the latter’s histone deacetylase inhibitor (HDACi) library and related intellectual property. The deal covers compounds for the potential treatment of patients suffering from Friedreich's ataxia, a rare neurological disorder.
Apart from the interesting pipeline, BioMarin has a strong product portfolio with four marketed products – Naglazyme, Kuvan, Aldurazyme and Firdapse. BioMarin co-markets Aldurazyme with Sanofi (SNY).
BioMarin currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biopharma space include Actelion Ltd. (ALIOF) and Medivation, Inc. (MDVN). Both stocks carry a Zacks Rank #1(Strong Buy).