Navigation Links
BioMarin Receives Orphan Drug Designation from the FDA for BMN-701 for the Treatment of Pompe Disease
Date:8/30/2010

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 www.BMRN.com. 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:
*Name:
*Comment:
*Email:
(Date:12/2/2016)... in the ECG Cables And ECG Leadwires Market owing to ... devices. On the other hand, the Asia-Pacific ... rate during the forecast period. The market players ... plc ( Ireland ), Koninklijke Philips N.V. ( ... ( China ), held major share of the ...
(Date:12/2/2016)... PUNE, India , December 2, 2016 /PRNewswire/ ... research report "In Vitro Diagnostics/IVD Market by Product ... Diagnostics, Hematology), Application (Diabetes, Oncology, Cardiology, Nephrology, Infectious ... the global market is valued at USD 60.22 ... grow at a CAGR of 5.5% during the ...
(Date:12/2/2016)... On Thursday, December 1st 2016, ... research, development and innovation in the biopharmaceutical industry at ... in the presence of Sergey Tsyb, Vice Minister of ... , Natalia Sanina, First Vice Chairman of the ... of Roszdravnadzor, National Service of Control in Healthcare, Sergey ...
Breaking Medicine Technology:
(Date:12/2/2016)... Milford, NJ (PRWEB) , ... December 02, 2016 , ... ... Nothing Water has some unique properties including its unmatched natural purity of just 6 ... as clean and crisp. , Nothing Water has been available in several ShopRite and ...
(Date:12/2/2016)... ... December 02, 2016 , ... Clarify Health ... has raised $6.0 million in an initial round of funding. The round was ... and their caregivers can receive far better care through the application of the ...
(Date:12/2/2016)... ... December 02, 2016 , ... ‘Tis the season for giving! Today, 20 ... the National Family Partnership and the Drug Enforcement Administration as part of the National ... the 10 winning schools who decorated their campuses with this year’s Red Ribbon Week ...
(Date:11/30/2016)... PALMYRA, Wisconsin (PRWEB) , ... November 30, 2016 ... ... inclusion in the 2016 Deloitte Wisconsin 75, an annual ranking and recognition of ... consecutive year on the list, having ranked from 2008-2016. In addition, Standard Process ...
(Date:11/30/2016)... ... November 30, 2016 , ... ... and stylish design wanted by today’s consumers at an affordable price, is now ... says the new watch is “a game changer” when it comes to the ...
Breaking Medicine News(10 mins):