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)... DUBLIN , November 26, 2015 ... has announced the addition of the  ... in the Global Cell Surface Testing ... Opportunities" report to their offering.  ... the addition of the  "2016 Future ...
(Date:11/26/2015)... November 26, 2015 ... addition of the  "2016 Future Horizons ... Therapeutic Drug Monitoring (TDM) Market: Supplier ... Emerging Opportunities"  report to their offering.  ... announced the addition of the  "2016 ...
(Date:11/26/2015)... 26, 2015 ... "Radioimmunoassay Market by Type (Reagents & ... Academics, Clinical Diagnostic Labs), Application (Research, Clinical ... 2020" report to their offering. ... addition of the "Radioimmunoassay Market by ...
Breaking Medicine Technology:
(Date:11/27/2015)... ... November 27, 2015 , ... ... toilets were," said an inventor from Hillside, N.J. "Many people catch diseases simply ... so that individuals will always be protected from germs." , He developed the ...
(Date:11/27/2015)... ANGELES, CA (PRWEB) , ... November 27, 2015 , ... MPWH, the No.1 Herpes-only dating ... of 30 (see Table 1-1 ). More than 3.7 billion people under the ... virus type 1 (HSV-1), according to WHO's first global estimates of HSV-1 infection . ...
(Date:11/27/2015)... (PRWEB) , ... November 27, 2015 , ... ... failing. Secura Consultants has prided itself for not only fulfilling the needs of ... protection solutions at an affordable price and providing top-tier customer service. However, there's ...
(Date:11/27/2015)... ... 2015 , ... Dr. Thomas Dunlap and Dr. Patrick Coleman ... with Emergency Medicine at St., Joseph Health System’s Santa Rosa Memorial Hospital , ... in similar ways and require time-critical intervention to avoid large area heart damage and ...
(Date:11/26/2015)... ... November 26, 2015 , ... Inevitably when people think Thanksgiving, they ... to buy during the Black Friday and Cyber Monday massage chair sales ... the Internet high and low to find the best massage chair deals, they can ...
Breaking Medicine News(10 mins):