Good News for BioMarin - Analyst Blog

BioMarin Pharmaceutical Inc. BMRN received encouraging news when the U.S. Food and Drug Administration (FDA) granted the company's marketed product Kuvan powder (for oral solution and tablets) a six-month pediatric exclusivity extension.

The FDA's decision resulted in a six-month extension of the product's marketing exclusivity period to Jun 2015, during which Kuvan's generic may not be cleared following its patent expiry. Kuvan is marketed for treating patients with phenylketonuria (PKU), a rare genetic disorder.

The U.S. regulatory body arrived at the decision to extend Kuvan's marketing exclusivity period on the basis of data from the studies submitted by the company. The submission followed a written request by the FDA to evaluate the use of the drug in pediatrics (from birth to 6 years old).

BioMarin stated in its press release that Kuvan in the form of tablets and powder for oral solution is the first and only treatment to be approved in the U.S. for PKU to bring down the level of blood phenylalanine (Phe) in patients suffering from hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive PKU. Kuvan should be used in combination with a Phe-restricted diet.

The bodies of patients affected with PKU are unable to process Phe, an amino acid found in many foods, leading to excessive Phe in the blood. The disorder can affect the brain, impair thinking and cause behavioral problems if not treated promptly.

The powdery form of Kuvan is more convenient for infants and children compared to tablets as it dissolves rapidly in water, apple juice or a small amount of soft food, thereby reducing chances of acidity. Kuvan is one of the most important products in BioMarin's product portfolio. Consequently, we are positive on the FDA's move to extend its marketing exclusivity period.

Apart from Kuvan, the other marketed products of the company are Naglazyme, Aldurazyme, Firdapse and Vimizim. BioMarin co-markets Aldurazyme with Sanofi SNY. Apart from the strong product portfolio, BioMarin has a robust pipeline with several data readouts expected this year.

BioMarin, a biopharmaceutical company, currently carries a Zacks Rank #4 (Sell). Better-ranked stocks in the sector include Regeneron Pharmaceuticals, Inc REGN and Alexion Pharmaceuticals, Inc. ALXN. While Regeneron carries a Zacks Rank #1 (Strong Buy), Alexion holds a Zacks Rank #2 (Buy).