What's Causing The Rally In BioMarin? CEO Answers

Shares of BioMarin Pharmaceutical Inc. BMRN have witnessed a spectacular rise in the last year, up by more than 67 percent. What's causing this rally?

Jean-Jacques Bienaime, BioMarin Pharmaceuticals CEO, was on CNBC Monday to answer that question and to discuss the new drugs that the company is developing. Bienaime also took the opportunity to discuss BioMarin's acquisition strategy.

Largest Pipeline Among Firms Focusing On Rare Disorders

"We have eight new molecular entities in clinical development today," Bienaime said. "We have the largest pipeline of any biotech company focusing on rare and ultra-rare disorders, and the most advanced is [...] Drisapersen for Duchenne Muscular Dystrophy."

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He continued, "We have a rolling NDA [new drug application] ongoing right now. We should complete the NDA filing at the end of next month, and this is against a very severe debilitating lethal disorder for which there is no approved therapy today. And Drisapersen, our product, is the only product that has breakthrough therapy designation in that space with the FDA."

Acquisition Strategy

Bienaime was asked that if BioMarin is focusing primarily on rare disorders, it perhaps would be looking to acquire other biotech firms in the same space and not rely solely on its own R&D. He replied, "That is correct. Although, we have a pretty powerful research and discovery organization. But we constantly look at other products potentially that could fit within our strategy.

"There are over 6,000 rare disorders in the world. So, there's plenty of opportunity to discover new products where the biology of the disease is very well understood, which is where we focus."