Navigation Links
FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS

NOVATO, Calif., Nov. 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the Food and Drug Administration (FDA) has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U. Also, in October 2009, the Committee for Medicinal Products for Human Use of the European Medicines Evaluations Agency adopted a positive opinion recommending approval of amifampridine phosphate for LEMS. If approved by the European Commission, amifampridine phosphate will be the first approved treatment for LEMS, thereby conferring orphan drug protection and providing ten years of market exclusivity in Europe.

"With its experience in the development and commercialization of orphan drugs, BioMarin is well-positioned to seek U.S. registration of amifampridine phosphate for LEMS, a serious and debilitating autoimmune disease often associated with small cell lung cancer," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We look forward to meeting with the FDA in early 2010 to determine the necessary regulatory path for amifampridine phosphate in the U.S. We are also preparing to launch the product in Europe in the first quarter of 2010, and will also evaluate the best development strategy for amifampridine phosphate in other indications in the U.S. and Europe."

About LEMS

Lambert Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disease with the primary symptoms of muscle weakness. Muscle weakness in LEMS is caused by autoantibodies to voltage gated calcium channels leading to a reduction in the amount of acetylcholine released from nerve terminals. The prevalence of LEMS is estimated at four to ten per million, or approximately 2,000 to 5,000 patients in the E.U. and 1,200 to 3,100 patients in the U.S. Approximately 50 percent of LEMS patients diagnosed have small cell lung cancer.

Patients with LEMS typically present with fatigue, muscle pain and stiffness. The weakness is generally more marked in the proximal muscles particularly of the legs and trunk. Other problems include reduced reflexes, drooping of the eyelids, facial weakness and problems with swallowing. Patients often report a dry mouth, impotence, constipation and feelings of light headedness on standing. On occasion these problems can be life threatening when the weakness involves respiratory muscles. A diagnosis of LEMS is generally made on the basis of clinical symptoms, electromyographic testing and the presence of autoantibodies against voltage gated calcium channels.

Current treatment of LEMS can consist of strategies directed at the underlying malignancy if one is present. Unfortunately, therapy of small cell lung cancer is limited and outcomes are generally poor. Immunosuppressive agents have been tried but success is limited by toxicity, and difficulty administering the regimens. A mainstay of therapy has been 3,4-DAP but its use in practice has been limited by the drug's availability. This problem will be addressed by the introduction of BioMarin's product.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three 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; and Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical development for the treatment of MPS IVA. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations of the development and potential approval of Huxley's 3,4-Diaminopyridine product for the treatment of LEMS. 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, particularly the pending decision by the European Commission on the Marketing Authorization Application for such product, our success in the commercialization of such product, if approved; 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 2008 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.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.


Investors                               Media
Eugenia Shen                            Susan Berg
BioMarin Pharmaceutical Inc.            BioMarin Pharmaceutical Inc.
(415) 506-6570                          (415) 506-6594

SOURCE BioMarin Pharmaceutical Inc.

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2009 PR Newswire.
All rights reserved

Related biology technology :

1. Mirus Bio Awarded Grants & Contracts of Over $5 Million
2. FDA Grants Orphan Drug Designation to TREANDA, an Investigational Treatment for Chronic Lymphocytic Leukemia
3. Anesiva Grants Specific-Use License of Its Needle-Free Drug Delivery Technology to Particle Therapeutics for Diabetes Drug
4. NIH grants $750,000 to develop device to determine temperature of neonates brain
5. Edwards Lifesciences Grants UC Irvine $5 Million to Establish the Edwards Lifesciences Center for Advanced Cardiovascular Technology
6. Radius Grants Major Pharmaceutical Company Exclusive Option to License BA058 for Osteoporosis
7. Poniard Pharmaceuticals Announces European Commission Grants Orphan Medicinal Product Designation to Picoplatin for Treatment of Small Cell Lung Cancer
8. CV Therapeutics Reports Option Grants Under Nasdaq Marketplace Rule 4350
9. NASDAQ Panel Grants Merge Technologies Additional Time to Regain Compliance
10. FDA Grants Resverlogix Approval to Commence a Phase 1 Clinical Trial for RVX-208
11. Maryland Stem Cell Commission Issues Nationwide Request for Applications for New Post-Doctoral Fellowship Grants
Post Your Comments:
(Date:11/25/2015)... , November 25, 2015 ... Report is a professional and in-depth study on ...      (Logo: ) , ... the industry including definitions, classifications, applications and industry ... for the international markets including development trends, competitive ...
(Date:11/24/2015)... , Nov. 24, 2015 Halozyme Therapeutics, Inc. (NASDAQ: ... in New York on Wednesday, December 2 ... Torley , president and CEO, will provide a corporate overview. ... York at 1:00 p.m. ET/10:00 a.m. PT . ... relations, will provide a corporate overview. --> th ...
(Date:11/24/2015)... -- Clintrax Global, Inc., a worldwide provider of clinical research services headquartered ... the company has set a new quarterly earnings record in Q3 ... posted for Q3 of 2014 to Q3 of 2015.   ... , with the establishment of an Asia-Pacific ... United Kingdom and Mexico , with ...
(Date:11/24/2015)... 2015 --> ... released by Transparency Market Research, the global non-invasive prenatal ... of 17.5% during the period between 2014 and 2022. ... Industry Analysis, Size, Volume, Share, Growth, Trends and Forecast ... market to reach a valuation of US$2.38 bn by ...
Breaking Biology Technology:
(Date:11/9/2015)... 2015 ... "Global Law Enforcement Biometrics Market 2015-2019" ... ) has announced the addition ... Market 2015-2019" report to their offering. ... ) has announced the addition of ...
(Date:11/2/2015)... Calif. , Nov. 2, 2015  SRI International ... million to provide preclinical development services to the National ... contract, SRI will provide scientific expertise, modern testing and ... variety of preclinical pharmacology and toxicology studies to evaluate ... --> The PREVENT Cancer Drug Development Program ...
(Date:10/29/2015)... 2015 Daon, a global leader in mobile ... a new version of its IdentityX Platform , ... America have already installed IdentityX v4.0 and ... FIDO UAF certified server component as an ... FIDO features. These customers include some of the largest ...
Breaking Biology News(10 mins):