Navigation Links
BioMarin Receives Orphan Drug Designation from the FDA for BMN-701 for the Treatment of Pompe Disease

NOVATO, Calif., Aug. 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease.  An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is expected to start in the first quarter of 2011.

"Receiving orphan drug designation from the FDA for BMN-701 is a significant milestone for our Pompe program.  As part of their assessment for designation, the FDA determined that BMN-701 is sufficiently different from alglusidase alfa (Myozyme/Lumizyme) to allow for a unique orphan designation.  For this reason, clinical superiority over alglusidase alfa will not be necessary to secure orphan exclusivity for BMN-701," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "This emphasizes our mission of developing innovative, products for orphan diseases with an unmet medical need.  We believe BMN-701 has the potential to possibly deliver more enzyme to lysosomes compared to traditional mannose-6-phosphate targeted approaches using the recently acquired GILT technology."  

About Pompe DiseasePompe disease, a lysosomal storage disorder, is a progressive degenerative disease of the heart muscle, diaphragm and skeletal muscle.  It is caused by a deficiency in the lysosomal enzyme acid alpha glucosidase which leads to the accumulation of glycogen in myocyte lysosomes and results in cell death.  The incidence is one in 40,000 births.  There are two main forms of Pompe disease: adult onset with an incidence of one in 57,000 births and infantile onset with an incidence of one in 138,000 births.  The current standard of care is Genzyme's Myozyme and Lumizyme.  Prognosis with standard of care is stabilization of the disease or minor improvements for the majority of adult onset patients.  

About BioMarinBioMarin 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 phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Other product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in clinical development for the treatment of MPS IVA and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking StatementThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations related to regulatory actions on BMN-701 and the development and efficacy of BMN-701. 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: the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities, results and timing of current and planned preclinical studies and clinical trials related to such product; our ability to successfully manufacture the product; 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 2009 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. 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.

BioMarin®, Naglazyme® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.

Firdapse™ is a trademark of BioMarin Huxley Ltd.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.Contact:Eugenia ShenBioMarin Pharmaceutical Inc.(415) 506-6570

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. BioMarin Partner AnGes MG, Inc. Submits BLA to Japanese Ministry of Health
2. BioMarin Announces FDA Approval for Kuvan
3. BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU
4. BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease
5. BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome
6. BioMarin Initiates Phase 2 Clinical Study of PEG-PAL in PKU
7. BioMarin Acquires Huxley Pharmaceuticals, Inc.
8. BioMarin to Present at the Oppenheimer Healthcare Conference
9. BioMarin Announces Third Quarter 2009 Financial Results
10. BioMarin to Acquire LEAD Therapeutics
11. BioMarin to Present at the Credit Suisse Healthcare Conference
Post Your Comments:
(Date:11/26/2015)... 2015 Research and Markets ( ... "2016 Future Horizons and Growth Strategies in the ... Country Segment Forecasts, Competitive Intelligence, Emerging Opportunities" ... --> --> This new 247-page ... therapeutic drug monitoring market, including emerging tests, technologies, ...
(Date:11/26/2015)... 2015 --> ... potential att använda SyMRI för att hitta ... patienter med multipel skleros (MS) eller hjärntumörmetastaser ... AB för att kunna använda SyMRI Research ... SyMRI kan man generera flera konstrastbilder från ...
(Date:11/26/2015)... , November 26, 2015 ... the "Self Administration of High Viscosity Drugs" ... ) has announced the addition of the ... report to their offering. --> Research ... addition of the "Self Administration of High ...
Breaking Medicine Technology:
(Date:11/27/2015)... ... 27, 2015 , ... The men and women on this ... in the country. They have overseen financial turnarounds, shown commitment to their community ... industry as a whole through their advocacy and professional efforts. , Becker's Hospital ...
(Date:11/27/2015)... ... November 27, 2015 , ... Indosoft Inc., developer ... incorporation of Asterisk 11 LTS (Long Term Support) into its Q-Suite 5.10 product ... Q-Suite 5.10 up-to-date with a version of Asterisk that will receive not only ...
(Date:11/26/2015)... Cambridge, ON (PRWEB) , ... November 26, 2015 ... ... availability of a real-time eReferral system for diagnostic imaging in the Waterloo region. ... mammography, BMD and Nuclear Medicine tests directly from their electronic medical record (EMR) ...
(Date:11/26/2015)... NE (PRWEB) , ... November 26, 2015 , ... Jobs ... searched by healthcare professionals and offered by healthcare staffing agency Aureus Medical Group ... during the month of October 2015 among those searching for healthcare jobs through the ...
(Date:11/26/2015)... ... November 26, 2015 , ... Pixel Film Studios ... titles with ProTrailer: Vintage. This newly styled ProTrailer pack comes with 30 all-new ... any font, giving users limitless opportunities to stylize and create designs quickly and ...
Breaking Medicine News(10 mins):