Press release from GlobeNewswire (a Nasdaq OMX company)
Vimizim Marketing Application Submitted to ANVISA in Brazil
Monday, July 01, 2013
Vimizim Marketing Application Submitted to ANVISA in Brazil06:00 EDT Monday, July 01, 2013
SAN RAFAEL, Calif., July 1, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that it has submitted the Vimizim Marketing Application (MA) to the Agência Nacional de Vigilancia Sanitaria (ANVISA), or the National Health Surveillance Agency Brazil. Assuming priority review status, based on orphan drug status in the U.S. and EU, an approval decision from ANVISA is anticipated in mid 2014.
"Considering that Brazil, along with the U.S., are expected to be the largest market opportunities for Vimizim, the timely submission in this market is an important milestone in our efforts to bring the first therapeutic option to patients with Morquio A Syndrome," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "Along with orphan drug designation in the U.S. and EU, priority review status in the U.S. and accelerated assessment status in the EU, we are making good progress on a global basis. In the coming months, we will continue to work closely with regulatory authorities and prepare for the launch of Vimizim worldwide."
About MPS IVA
Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine-6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.
The rate of incidence of MPS IVA is as yet unconfirmed and varies among different populations but estimates vary between 1 in 200,000 live births and 1 in 250,000 live births. The estimated prevalence is approximately 3,000 patients in the developed world. Based on knowledge of the worldwide distribution of the MPS VI market and the more than 1,300 identified MPS IVA patients worldwide, the company estimates that approximately 20 percent of patients are in North America (15 percent in the U.S.) and approximately 50 percent of patients are in EUMEA.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include BMN-110 (elosulfase alfa), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase III clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: expectations regarding the MA filing for Vimizim with ANVISA; the potential outcome of the review of such filing; and the possible approval of such product candidate. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned clinical trials of its product candidates; the nature of the ANVISA's questions associated with the MA and BioMarin's ability to timely respond to those questions; BioMarin's ability to continue to meet the requirements for accelerated assessment; ANVISA's compliance with it internal review guidelines; the content and timing of decisions by ANVISA; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2012 Annual Report on Form 10-K, as amended, and the factors contained in BioMarin's reports on Form 8-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
Vimizim™ is our trademark, and BioMarin®, Naglazyme®, Kuvan®, Firdapse® are registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
CONTACT: Investors: Eugenia Shen BioMarin Pharmaceutical Inc. (415) 506-6570 Media: Debra Charlesworth BioMarin Pharmaceutical Inc. (415) 455-7451