Navigation Links
FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS
Date:11/30/2009

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
'/>"/>

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

Page: 1 2 3 4

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:
*Name:
*Comment:
*Email:
(Date:7/30/2014)... Sales Horizons, a leader in sales ... training programs for companies engaged in complex B2B sales. ... thousands of salespeople over 25 years in market-leading Fortune ... be customized to address the unique sales challenges of ... training program consists of two parts: Mastering the ...
(Date:7/30/2014)... 30, 2014 BCC Research ( http://www.bccresearch.com ... for Enzymes in Industrial Applications , the global market ... $7.1 billion by 2018, registering a five-year compound annual ... to be recorded in the detergent enzyme segment with ... Enzyme technology has influenced almost every sector of industrial ...
(Date:7/30/2014)... July 30, 2014  Dyadic International, Inc. ("Dyadic") ... patented and proprietary technologies are used to discover, ... for the bioenergy, bio-based chemical, biopharmaceutical and industrial ... "Tom" Dubinski as Vice President and Chief Financial ... financial and information technology operations for Dyadic and ...
(Date:7/30/2014)... MARLBOROUGH, Mass. , July 30, 2014 ... RXII ), a biotechnology company focused on ... major unmet medical needs using RNA-targeted technologies, ... study (RXI-109-1402) with RXI-109, for the reduction ... scar revision surgery, has been initiated. ...
Breaking Biology Technology:Sales Horizons Launches New Blended Sales Training Programs to Companies Engaged in Complex B2B Sales 2Sales Horizons Launches New Blended Sales Training Programs to Companies Engaged in Complex B2B Sales 3Global Market for Industrial Enzymes to Reach Nearly $7.1 Billion by 2018; Detergent Enzyme Market to Record Maximum Growth 2Global Market for Industrial Enzymes to Reach Nearly $7.1 Billion by 2018; Detergent Enzyme Market to Record Maximum Growth 3Thomas Dubinski joins Dyadic as Chief Financial Officer 2Thomas Dubinski joins Dyadic as Chief Financial Officer 3Thomas Dubinski joins Dyadic as Chief Financial Officer 4RXi Pharmaceuticals Expands Clinical Program with Initiation of Third Phase 2a Study with RXI-109 for Treatment of Hypertrophic Scars 2RXi Pharmaceuticals Expands Clinical Program with Initiation of Third Phase 2a Study with RXI-109 for Treatment of Hypertrophic Scars 3RXi Pharmaceuticals Expands Clinical Program with Initiation of Third Phase 2a Study with RXI-109 for Treatment of Hypertrophic Scars 4RXi Pharmaceuticals Expands Clinical Program with Initiation of Third Phase 2a Study with RXI-109 for Treatment of Hypertrophic Scars 5
... 7, 2011 Reportlinker.com announces that a new ... Industrial Biotechnology China News 1103 ... The Chinese central government was reported to continue ... the Twelfth Five-Year (2011-2015) Plan. This act is ...
... and industrial research scientists and engineers, as well ... encounter a steep learning curve when developing common ... book just published by Springer promises to be ... The new MEMS Materials and Processes Handbook is ...
... The U.S. Food and Drug Administration today approved ... tool to treat brain aneurysms without performing open surgery. ... An aneurysm is a weak, enlarged area in an ... blood pressure can cause it to rupture, which can ...
Cached Biology Technology:Reportlinker Adds Industrial Biotechnology China News 1103 2Reportlinker Adds Industrial Biotechnology China News 1103 3MEMS Materials and Processes Handbook -- a comprehensive, practical resource 2FDA Approves New Device to Treat Brain Aneurysms 2FDA Approves New Device to Treat Brain Aneurysms 3
(Date:7/30/2014)... increase in Impact Factors for its molecular bioscience ... (Thomson Reuters, 2014) show that Clinical Science ... Biochemistry , Biochemical Society Transactions and ... their Impact Factors. The journals are owned by ... Limited. , 2014 Impact Factors (2013 Impact Factor ...
(Date:7/30/2014)... State University,s College of Engineering recently received a ... the project, "Nanoparticle-directed synthesis of organic nanorods." ... is the creation and utilization of functional materials, ... A major bottleneck in scaling up nanotechnology is ... functional materials into one device. A research team ...
(Date:7/30/2014)... into a multitude of cells types. Researchers would like to ... of mature cells that make up the organs and other ... be long chains of sugars that dangle from proteins on ... of California, San Diego, has created synthetic molecules that can ... easily manipulated to direct the process, they report in the ...
Breaking Biology News(10 mins):Across-the-board Impact Factor increases for Portland Press Limited 2NSF grant to Wayne State supports new concept for manufacturing nanoscale devices 2
... , This news release is available ... Medical implants, complex interfaces between brain and machine or ... great potentials, but also give rise to major ethical concerns. ... technischer Systeme mit Lebewesen" (The Chemistry of Cyborgs Interfacing ...
... cells scientists at Brigham and Women,s Hospital and MIT ... following transplantation, potentially making cell therapies, hundreds of which ... more functional and efficient. Associate Professor Jeffrey Karp, ... issue of Nature Protocols how to load ...
... , Jan. 10, 2014   BioNano Genomics announced today ... present at the 32nd Annual J.P. Morgan Healthcare Conference in ... p.m. PST.  About BioNano Genomics ... delivering an altogether better way of gaining a fully informed understanding ...
Cached Biology News:The cyborgs era has started 2The cyborgs era has started 3Harvard scientists control cells following transplantation, from the inside out 2
... Gene Expression (SAGE) is a powerful ... a cost-effective outsourcing solution for sequencing ... Agencourt's SAGE sequencing service provides our ... long Phred20 read lengths (40 SAGE ...
... Sciences can provide custom microplate coating using advanced automated high ... of plate formats. We can also accommodate virtually any lot ... 0.5-320 L):, , 8 ... well microplate, 384 well microplate, ...
... ) The antibody is preadsorbed to remove any ... buffered aqueous glycerol solution A solution ... 2+ and Ca 2+ ), pH 7.3 ... (IgG, protease free) and 0.05% sodium azide A ...
... Cytometry The Guava EasyCyte system is ... multiparameter cytometer with built-in 96-well microplate sampling ... plates of cells and compounds quickly and ... tubes or having the carryover issues found ...
Biology Products: