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:10/22/2014)... NEW YORK , Oct. 22, 2014 ... Diabetes Federation, approximately 400 million people worldwide are living ... 552 million people by 2030.  The global market for ... In 2012 approximately 330,000 people worldwide died from pancreatic ... of death due to cancer in the ...
(Date:10/22/2014)... HONG KONG , Oct. 22, 2014  aTyr ... today that rare disease expert John C. McKew ... Dr. McKew brings more than two decades of expertise ... National Institutes of Health, Wyeth Research and Genetics Institute, ... lead aTyr,s efforts to expand and translate its novel ...
(Date:10/20/2014)... Oct. 20, 2014 Mapp Biopharmaceutical,s valiant ... antibody therapeutic to fight the Ebola outbreak will ... time-consuming the production of pharmaceuticals can be, according ... said that while some may be taken aback ... those with industry knowledge are well aware of ...
(Date:10/20/2014)... Oct. 20, 2014 Asterias Biotherapeutics, Inc. ... has signed a Notice of Grant Award (NGA) ... effective October 1, 2014.  The NGA provides for ... the release of additional grant funds pursuant to ... for clinical development of Asterias, product, AST-OPC1. The ...
Breaking Biology Technology:Nuvilex Brief Analyst Report: Thinking Outside the Box by BrokerBank Securities, Inc. 2aTyr Pharma Appoints John C. McKew, Ph.D., as Vice President, Research 2aTyr Pharma Appoints John C. McKew, Ph.D., as Vice President, Research 3Kalorama: ZMapp Highlights Need For Faster Biopharmaceutical Production 2Kalorama: ZMapp Highlights Need For Faster Biopharmaceutical Production 3Asterias Biotherapeutics Announces Notice of Grant Award with CIRM for Phase 1/2a Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury 2Asterias Biotherapeutics Announces Notice of Grant Award with CIRM for Phase 1/2a Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury 3Asterias Biotherapeutics Announces Notice of Grant Award with CIRM for Phase 1/2a Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury 4
... BioLife Solutions, Inc.,(OTC Bulletin Board: BLFS), ... hypothermic storage and cryopreservation media products for ... of its new quarterly,newsletter BioPreservation Today, a ... and processes, to maximize the yield and,function ...
... Md., Jan. 26 MaxCyte, Inc., the pioneer ... the presentation of the MaxCyte(R) STX(TM) Scalable Transfection ... Session, a joint program of the Association for ... Group (LRIG). The Rapid Fire Innovation Session showcases ...
... as subcontractor to Battelle for submission of proposals ... 26 Corgenix Medical Corporation (OTC Bulletin Board: ... test kits, has announced a collaboration with Battelle, ... whose business focus includes national security. Under the ...
Cached Biology Technology:BioLife Solutions Launches BioPreservation Today(R) Industry Newsletter 2BioLife Solutions Launches BioPreservation Today(R) Industry Newsletter 3The MaxCyte(R) STX(TM) Scalable Transfection System to be Presented at the Rapid Fire Innovation Session at LabAutomation2009 2Corgenix Signs Collaboration With Battelle 2Corgenix Signs Collaboration With Battelle 3
(Date:10/19/2014)... electricity access in India over the last 30 ... carbon emissions growth during that time, according to ... Nature Climate Change ., "Energy access is fundamental ... of life, including education, communication, and health," says ... , While increased energy access is widely agreed ...
(Date:10/18/2014)... sample of patients with undiagnosed, suspected genetic conditions, a ... a higher molecular diagnostic yield than traditional molecular diagnostic ... . The study is being released to coincide with ... Exome sequencing, which sequences the protein­coding region of the ... present in a cell or organism), has been rapidly ...
(Date:10/17/2014)... for the first time how bacteria can grow directly ... the opportunity to get tremendous insights into bacteria behavior ... discovered the bacterial growth in chronic lung infections among ... by the immune cells. The researchers discovered the immune ... bacteria, forcing the bacteria to switch to a much ...
Breaking Biology News(10 mins):Improved electricity access has little impact on climate change 2Study examines type of exome sequencing and molecular diagnostic yield 2Study examines type of exome sequencing and molecular diagnostic yield 3Scientists opens black box on bacterial growth in cystic fibrosis lung infection 2
... is available in German . , Four ... year,s Heinz Maier-Leibnitz Prize, Germany,s top research award for young ... the prize, which has been awarded annually by the Deutsche ... of Education and Research since 1977, that the majority of ...
... is available in German . The gene ... by cell division. It causes the production of a protein ... to 15 % of all human genes. When this gene ... is inhibited. Thereby the gene plays a decisive role in ...
... is a familiar phenomenon, even if the phrase is not: ... it around the block. After jogging regularly for a few ... three. With regular exercise, the body adapts, becoming fitter and ... oxygen to the muscles. The muscles get stronger, and so ...
Cached Biology News:Heinz Maier-Leibnitz Prizes 2009: Six young researchers recognized for outstanding achievements 2Heinz Maier-Leibnitz Prizes 2009: Six young researchers recognized for outstanding achievements 3Heinz Maier-Leibnitz Prizes 2009: Six young researchers recognized for outstanding achievements 4Heinz Maier-Leibnitz Prizes 2009: Six young researchers recognized for outstanding achievements 5Symposium to look at genetic basis of exercise 2Symposium to look at genetic basis of exercise 3
Human/Rat Neurogenin-2 MAb (Clone 7G4) Protein Family: Neural Crest Cell Markers, Transcription Factors...
Rat CINC-2 alpha/beta Affinity Purified Polyclonal Ab...
Monkey IL-2 ELISPOT Kit with white/opague microtiter plates...
Mouse TREM-2 Affinity Purified Polyclonal Ab...
Biology Products: