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

1

GOOD

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:5/23/2013)... , May 23, 2013 Pressure BioSciences, ... that the Company will host a teleconference to discuss ... a business update. The teleconference information is provided below: ... a.m. Eastern Daylight Time (EDT) Dial-in: (800) ... (302) 607-2001 (Int,l) Passcode: VS22823 For those ...
(Date:5/23/2013)... SynCardia Systems, Inc. ( http://www.syncardia.com ), manufacturer of ... CE (Europe) approved Total Artificial Heart, announced today that ... than 380,000 visitors annually, is featuring the SynCardia ... portable driver as part of its $3 million Bodyworks ... kind. , “BodyWorks combines the latest technology with ...
(Date:5/23/2013)... St. Louis, Missouri (PRWEB) May 23, 2013 ... design and manufacture of complete thermal systems, recently ... more than 90 years of experience in designing ... most complete selection of products to serve diverse ... lead-time options is featured at the beginning of ...
(Date:5/22/2013)... (PRWEB) May 22, 2013 Industry ... Alexandria, VA for the Oak Ridge National ... will take place at the Hilton Alexandria Mark ... Biodetection Technologies 2013 conference on June 18 ... Participating: , AdSave, American Elements, Applied Research Associates, ...
Breaking Biology Technology:Pressure BioSciences, Inc. to Discuss First Quarter 2013 Financial Results and Provide Business Update 2SynCardia Total Artificial Heart Featured in $3 Million Exhibition at Glasgow Science Centre 2SynCardia Total Artificial Heart Featured in $3 Million Exhibition at Glasgow Science Centre 3Watlow Introduces Its New Industrial Heater Catalog 2Biodetection Technologies 2013 and Oak Ridge National Lab’s Biosurveillance Symposium to Bring Together Leading Organizations in Alexandria, VA from June 17-19, 2013 2Biodetection Technologies 2013 and Oak Ridge National Lab’s Biosurveillance Symposium to Bring Together Leading Organizations in Alexandria, VA from June 17-19, 2013 3Biodetection Technologies 2013 and Oak Ridge National Lab’s Biosurveillance Symposium to Bring Together Leading Organizations in Alexandria, VA from June 17-19, 2013 4Biodetection Technologies 2013 and Oak Ridge National Lab’s Biosurveillance Symposium to Bring Together Leading Organizations in Alexandria, VA from June 17-19, 2013 5
... Osteotech, Inc.,(Nasdaq: OSTE ), a leader in ... announced today that Sam Owusu-Akyaw, Osteotech,s,President and Chief ... Orthopedics Conference at the Mission Bay Conference Center ... to present at 3:10 p.m.,Pacific Time on Tuesday, ...
... second month in a row,the capital markets succumbed to fears ... the month the Dow dropped over 300 points after,oil prices ... these,fears, biotech was dragged down with the Burrill Biotech Select ... Dow, which closed down,3%., "The biotech sector started 2008 ...
... engines have been selected, - New environmental partnership with Rolls Royce ... ... burn, SOUTH NORWALK, Conn., March 3 Virgin Atlantic, ... 1000 engines to power its Boeing 787-9 fleet of aircraft., (Logo: ...
Cached Biology Technology:Biotech Drops Amid Tough Markets in February 2Biotech Drops Amid Tough Markets in February 3Virgin Atlantic Selects Rolls-Royce Engines to Power Its 787 Fleet in a Deal Worth $2.6BN 2Virgin Atlantic Selects Rolls-Royce Engines to Power Its 787 Fleet in a Deal Worth $2.6BN 3Virgin Atlantic Selects Rolls-Royce Engines to Power Its 787 Fleet in a Deal Worth $2.6BN 4
(Date:5/22/2013)... The feared Legionella pneumophila bacteria is responsible ... pneumonia. In order to infect us, this pathogen has ... and go unnoticed in our cells, thus avoiding these ... the Basque biosciences research centre, CIC bioGUNE, in which ... the USA and the National Supercomputation Centre in Barcelona ...
(Date:5/22/2013)... NEW YORK, May 22, 2013. Plastic additives known ... just about everywhere: They turn up in flooring, ... andaccording to the Centers for Disease Control and ... harmless, phthalates have come under increasing scrutiny. A ... phthalates (which can leech from packaging and mix ...
(Date:5/21/2013)... the nation,s land and water resources could likely support ... 25 billion gallons of algae-based fuel a year in ... , The findings come from an in-depth look at ... significant amounts of algae in large, specially built shallow ... issue of Environmental Science and Technology , published ...
Breaking Biology News(10 mins):Mechanism discovered which aids Legionella to camouflage itself in the organism 2Study links chemicals widely found in plastics and processed food to elevated blood pressure in children and teens 2Going green: Nation equipped to grow serious amounts of pond scum for fuel 2Going green: Nation equipped to grow serious amounts of pond scum for fuel 3Going green: Nation equipped to grow serious amounts of pond scum for fuel 4
... WI, November 16, 2009 -- A USDOE and USDA ... idle cropland, and cropland pasture could be converted from ... as switchgrass, from which biomass could be harvested for ... a perennial grass monoculture from which substantial quantities of ...
... University of New Hampshire microbiologist Louis Tisa ... understanding of the actinorhizal plants, widespread woody plants ... The United States Department of Agriculture ... and biomedical sciences, $399,000 to explore the symbiotic ...
... cardiologist Tzung "John" Hsiai hopes to develop a ... requiring immediate surgery from chronic problems manageable with ... by catheters inserted into the arteries feeding the ... surface ("lumen") of these blood vessels, often revealing ...
Cached Biology News:Switchgrass produces biomass efficiently 2UNH prof. receives nearly $500,000 to research environmentally significant plants 2Stable plaque or heart attack plaque? USC researcher builds new sensor to tell which is which 2
... Dr. P Set from different human ... More organs maybe available upon request. ... items from the whole set, and ... price. All Dr. P Set ...
... of buffer and fixative results in ... In Vivo Manual Perfusion Fixation System ... buffer and fixative results in complete ... and clean--Complete system with everything down ...
Mouse IL-31 RA Biotinylated Affinity Purified PAb...
... The steadylite plus assay system ... assay sensitivity, Designed for batch-processing ... for higher density microplates such ... Odor free, Convenient storage conditions ...
Biology Products: