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:10/1/2014)... Oct. 1, 2014  Discovery Laboratories, Inc. (Nasdaq: ... awarded a Phase II Small Business Innovation Research ... from the National Institute of Allergy and Infectious ... (NIH) to support the development of the Company,s ... countermeasure to mitigate acute and chronic/late-phase radiation-induced lung ...
(Date:10/1/2014)... , Oct. 1, 2014  Spherix Incorporated (Nasdaq: ... fostering and monetization of intellectual property, announced today ... VTech and Uniden in the U.S. District Court ... has been rescheduled for November 10, ... 2, 2014.  The Court Order setting the Markman ...
(Date:10/1/2014)... (PRWEB) September 30, 2014 The participants ... designs, but to spending their own time adjusting, tweaking ... their complex designs were ready for judging. , “I ... design firm thought leader. “The entries showed a ... the physical characteristics of the print medium and the ...
(Date:10/1/2014)... Washington, USA (PRWEB) September 30, 2014 ... yet efficient arrangements that have evolved in nature over ... biomimetic and bioinspired materials for applications in biophotonics ... Biomedical Optics . The journal is published by SPIE, ... SPIE Digital Library. Several of the peer-reviewed articles are ...
Breaking Biology Technology:Discovery Labs Receives SBIR Grant Valued up to $3.0 Million to Support Development of Aerosolized KL4 Surfactant to Address Radiation-Induced Lung Injury 2Discovery Labs Receives SBIR Grant Valued up to $3.0 Million to Support Development of Aerosolized KL4 Surfactant to Address Radiation-Induced Lung Injury 3Markman Hearing Date for VTech and Uniden Litigation Now Set for November 10, 2014 2Array Architects Challenges Staff to Create with 3D Printer 2Biomimetic and bioinspired materials enable new healthcare options, reports Journal of Biomedical Optics 2Biomimetic and bioinspired materials enable new healthcare options, reports Journal of Biomedical Optics 3
... Conference Call to be held on May 15, 2008 ... 8:00 am ... April 29 /Xinhua-PRNewswire-FirstCall/ -- 3SBio Inc.,(Nasdaq: SSRX ), ... and marketing biopharmaceutical,products, today announced that it will release ...
... real steps towards building a microscopic device that can ... Writing in the peer-reviewed publication, International Journal of Nanomanufacturing ... a nanoassembler. , In his 1986 book, The Engines ... aim of nanotechnology - to create an assembler, a ...
... Emisphere,Technologies, Inc. (Nasdaq: EMIS ) announced that ... the NASDAQ Stock Market (the "NASDAQ Letter"),advising that, ... Company,s,market value of listed securities had been below ... NASDAQ Global Market pursuant to,NASDAQ Marketplace Rule 4450(b)(1)(A). ...
Cached Biology Technology:3SBio Inc. Schedules 2008 First Quarter Earnings Release on Wednesday, May 14, 2008 2Are nanobots on their way? 2Are nanobots on their way? 3Emisphere Reports Receipt of Notification from NASDAQ Regarding Non-Compliance with Continued Listing Requirements 2Emisphere Reports Receipt of Notification from NASDAQ Regarding Non-Compliance with Continued Listing Requirements 3
(Date:9/30/2014)... the environmental effects of moorland burning is published today ... with the aim of relieving tensions on both sides ... EMBER (Effects of Moorland Burning on the Ecohydrology of ... moorland, which is practised predominantly to support red grouse ... peat hydrology, peat chemistry and physical properties, river water ...
(Date:9/30/2014)... have in common? Unlike most eukaryotic multicellular organisms ... these organisms are all polyploid, meaning they have three ... 3 and 4 sets of chromosomes, respectively, and strawberries ... fact most plant species are polyploid. Polyploidy, or genome ... only recently, with the development of molecular tools, has ...
(Date:9/30/2014)... team of environmental scientists and engineers, led by ... a three-year, $5.25 million grant from the U.S. ... Research to make a direct positive impact on ... and disposal of radioactive contaminants. , "Understanding the ... dealing with environmental aspects of nuclear technologies is ...
Breaking Biology News(10 mins):Grouse moor burning causes widespread environmental changes 2Gene doubling shapes the world: Instant speciation, biodiversity, and the root of our existence 2Gene doubling shapes the world: Instant speciation, biodiversity, and the root of our existence 3Gene doubling shapes the world: Instant speciation, biodiversity, and the root of our existence 4Researchers get $5.25 million to advance nuclear technologies in South Carolina 2
... image of fossil gliding reptile Mecistrotrachelos apeoros. Curved foot ... of a previously unknown, 220 million-year-old long-necked, gliding reptile ... an industrial-size CT scanner at Penn State's Center for ... small creatures are now known. , The new ...
... at the University of Missouri-Columbia are studying the status ... in the state during the 18th and 19th centuries, ... the late 19th century. The results of the MU ... Conservation to help manage Missouri’s black bear population. , ...
... enough to keep an avian marriage together for the Nazca ... the coast of Ecuador. , Many Nazca booby females switch ... Forest University study scheduled for publication in the journal Proceedings ... , This is surprising because there is an advantage to ...
Cached Biology News:CT scan reveals ancient long-necked gliding reptile 2MU researchers to study the status of black bears in Missouri 2Study investigates 'divorce' among Galapagos seabirds 2
For microscopy...
... that enables fully automated hybridization of microarrays ... has been designed to offer researchers improved ... to perform a wide range of applications. ... range 485C; denaturations of up to 99C. ...
... microarray slide processing offering reproducibility, reliability ... to miniaturization, automation and massive parallel ... tool with many applications in both ... range from gene expression profiling, gene ...
... The user-friendly Eppendorf Thermomixer R offers ... that require shaking, heating, and cooling. ... expanded its application and temperature control ... each accommodate 24 micro test tubes, ...
Biology Products: