BioMarin Pharmaceuticals acquired the Dutch company Prosensa for $680 million in December. Prosensa's lead product, an RNA therapeutic (exon-skipping) for muscular dystrophy, has a checkered past. Once part of a partnership arrangement with GlaxoSmithKline, the drug was returned to Prosensa in January 2014 after disappointing phase 3 clinical trial results. However, Prosensa revived the drug program in 2014, and filed an new drug application with the US Food and Drug Administration (FDA) for accelerated approval in October with encouraging results in a small phase 2 trial. The FDA signaled its interest in the drug by granting it breakthrough drug status in 2013. BioMarin CEO was quoted as saying his company has experience with shepherding drugs that require “interpretation” through the regulatory process.
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