BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Announced Today the Stock is Halted
Nov 24, 2015

SAN RAFAEL, Calif., Nov. 24, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that NASDAQ has halted trading of the company's stock.  The Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) of the U.S. Food and Drug Administration (FDA) is meeting today to review BioMarin's New Drug Application (NDA) for Kyndrisa, an investigational antisense oligonucleotide drug candidate for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

The PCNS advisory meeting is scheduled for November 24 at 8:00 a.m. to 5:30 p.m. EST. The briefing materials and webcast information can be found on the FDA website at:

The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the Kyndrisa NDA is December 27, 2015.

About Duchenne Muscular Dystrophy

Changes in the dystrophin gene (mutations) that lead to the near absence of dystrophin protein result in the most severe form of dystrophin deficient muscular dystrophy, Duchenne muscular dystrophy, also known as just Duchenne.  Boys living with Duchenne experience progressive muscle weakness, causing serious medical complications including serious heart or respiratory-related complications, resulting in death in early adulthood.

Primarily affecting boys, Duchenne affects approximately 1 in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood.  There is currently no FDA approved therapy designed specifically to treat Duchenne.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of five commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit

Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558

Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451