How Promising is BioMarin’s Late-Stage Pipeline?



Brineura: Late stage pipeline product

BioMarin Pharmaceutical’s (BMRN) late-stage pipeline includes cerliponase alfa. The company’s application for cerliponase alfa, an enzyme replacement therapy, is under review by the FDA for the treatment of CLN2 disease.

CLN2 is a fatal neurodegenerative disease. The children affected by it lose the ability to walk and talk by six years old. The Prescription Drug User Fee Act date for the drug is April 27, 2017. Cerliponase alfa holds priority review status and breakthrough therapy designation from the FDA. 

valuation catalysts in 2H16
The European Medicines Agency (or EMA) has started the formal review process for cerliponase alfa, and its decision is expected by 3Q17. Cerliponase alfa holds an orphan drug designation from the EMA. 

Other companies that operate in the rare disease space include Alexion Pharmaceuticals (ALXN), Vertex Pharmaceuticals (VRTX), and Sanofi (SNY) through its subsidiary, Genzyme.

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Pegvaliase is indicated for the lowering of blood phenylalanine levels in PKU (phenylketonuria) patients. You can read details about the drug in What Are BioMarin’s Products for Treating Phenylketonuria? BioMarin’s existing drugs coupled with Brineura and pegvaliase are expected to drive BMRN’s total revenue across $2 billion by 2020.

If BioMarin’s two late-stage products are approved, its share price could rise. Although the company hasn’t yet crossed $1 billion in revenue, its market capitalization stood at $16.6 billion on September 26, 2016, indicating investors’ belief in the company’s products.

To gain exposure to the rapidly growing BioMarin, you can invest in the PowerShares QQQ Trust, Series 1 ETF (QQQ). The fund holds 0.28% of its total assets in BioMarin.

In the next article, we’ll discuss BMRN’s early stage pipeline.


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