Navigation Links
Kuvan Receives Positive Opinion From CHMP for European Approval
Date:9/25/2008

ial treatment option for approximately 30 percent to 50 percent of the estimated 50,000 identified PKU patients in the developed world.

Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan has received seven years of market exclusivity in the United States. In November 2007, Merck Serono submitted a Marketing Authorization Application (MAA) to the EMEA for sapropterin dihydrochloride as an oral treatment for patients suffering from hyperphenylalaninemia (HPA) due to PKU or BH4 deficiency. If approved in the EU, it will receive 10 years of market exclusivity for this indication.

About PKU

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase. PAH is required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures, tremors, and limited cognitive ability. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients under the age of 40 in developed countries have been diagnosed at birth. To learn more about PKU, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

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(R) (galsulfase) for mucop
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2008 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related biology technology :

1. Kuvan, Naglazyme and Aldurazyme Data to Be Presented at the 57th Annual Meeting of the American Society of Human Genetics
2. Exclusive Rights to Kuvan(TM) Data Licensed to Asubio Pharma in Japan
3. Corgenix Receives Third U.S. Patent on AtherOx(R) Technology
4. Sustainable Oils Receives Regulatory Approval for Feeding Camelina Meal in Livestock Diets
5. U.S. Genomics Inc. Receives $4.5 Million Investment from BD
6. AdvanDx Receives FDA 510(k) Clearance for Yeast Traffic Light PNA FISH(TM)
7. PLC Systems Receives Delisting Notice From American Stock Exchange
8. Nephros Receives Non-Compliance Notification from AMEX
9. China Biologic Receives SFDA Approval for Clinical Trial of Human Coagulation Factor VIII
10. Barr Receives Approval for Generic Version of Razadyne(R) ER Capsules
11. Intact Medical Corporations Intact(R) Excise XL Receives 510(K) Marketing Clearance from the FDA
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/1/2014)... BOSTON, MA (PRWEB) September 30, 2014 ... company, SoundConnect announces four new-hosted collaboration ... innovative bundles deliver high quality conferencing experiences for ... collaboration packages to choose from, businesses have optimized ... bundled solutions include audio only minutes ...
(Date:10/1/2014)... Industry leaders from retail ... around the theme, ,Commercial Opportunities in the Era of ... Elsevier , a world-leading provider of scientific, ... a gathering of some of the industry,s most prominent ... today and in the future. The Elsevier ...
(Date:9/30/2014)... YORK , Sept. 30, 2014 ... - 2014 Summary Global Markets Direct,s, ... - 2014, provides an overview of the Pacific ... This report provides comprehensive information on the ... LLC,s, complete with comparative analysis at various stages, ...
(Date:9/30/2014)... Proove Biosciences , the commercial and research leader ... genetic test built upon research from their published P.A.I.N. ... provide physicians with an objective way to understand theirs ... between individuals. With this new test, physicians will be ... predisposition that will affect their pain tolerance. , The ...
Breaking Biology Technology:SoundConnect Launches New Audio, Web and Video Bundles 2Elsevier Healthcare and Pharmaceutical Economics Conference 2014 To Convene Industry Leaders to Discuss Top Healthcare Business Issues 2Elsevier Healthcare and Pharmaceutical Economics Conference 2014 To Convene Industry Leaders to Discuss Top Healthcare Business Issues 3Pacific Northwest Biotechnology, LLC - Product Pipeline Review - 2014 2Pacific Northwest Biotechnology, LLC - Product Pipeline Review - 2014 3Proove Biosciences is Now Offering the Proove Pain Perception Genetic Test to Help Physicians Assess Patient Pain Tolerance Levels 2Proove Biosciences is Now Offering the Proove Pain Perception Genetic Test to Help Physicians Assess Patient Pain Tolerance Levels 3
... ATLANTA, April 1, 2008 Piedmont Heart Institute ... 20-member cardiovascular,physician practice, Cardiology of Georgia, P.C. (COG), ... 1, 2008. With eight,locations including Buckhead, Canton, Fayetteville ... practice under the Piedmont Heart Institute and an,important ...
... April 1, 2008 ,Neurobiological Technologies, Inc. (NTI(R)) (Nasdaq: ... M. Loar has been appointed Chief,Financial Officer. Mr. Loar ... Loar joins the company from Osteologix, where he has ... a proven track record as a chief financial officer, ...
... LONDON, April 1, 2008 Plethora Solutions Holdings ... for the,treatment and management of urological disorders, today ... financing agreement with Paul Capital,Healthcare. Through this agreement, ... with $15 million payable upon signature and a ...
Cached Biology Technology:Piedmont Heart Institute Welcomes Cardiology of Georgia 2Piedmont Heart Institute Welcomes Cardiology of Georgia 3Piedmont Heart Institute Welcomes Cardiology of Georgia 4Neurobiological Technologies Appoints Matthew M. Loar as Chief Financial Officer 2Neurobiological Technologies Appoints Matthew M. Loar as Chief Financial Officer 3Plethora and Paul Capital Healthcare sign a $25 million revenue interest financing agreement with an option for an additional $3 million equity investment 2Plethora and Paul Capital Healthcare sign a $25 million revenue interest financing agreement with an option for an additional $3 million equity investment 3Plethora and Paul Capital Healthcare sign a $25 million revenue interest financing agreement with an option for an additional $3 million equity investment 4
(Date:9/30/2014)... Ocean Health Index rates the Earth,s oceans at ... addition, for the first time, the report assessed ... national jurisdiction (high-seas areas) all critical regions ... providing sustainable food sources. , In the third ... by scientists from UC Santa Barbara,s National Center ...
(Date:9/30/2014)... Calif. The National Institutes of Health (NIH) ... today to develop an electrode array system that ... brain works through unprecedented resolution and scale. , ... to support President Obama,s BRAIN (Brain Research ... research effort to revolutionize our understanding of the ...
(Date:9/30/2014)... drinking water may increase the risk of stillbirth ... led by a Boston University School of Public ... journal Environmental Health , compared 1,091 PCE-exposed ... in Cape Cod, Ma., where water was contaminated ... by the installation of vinyl-lined asbestos cement pipes. ...
Breaking Biology News(10 mins):Rating the planet's oceans 2Rating the planet's oceans 3NIH taps lab to develop sophisticated electrode array system to monitor brain act 2NIH taps lab to develop sophisticated electrode array system to monitor brain act 3Contaminated water linked to pregnancy complications, BU study finds 2
... - Rainfall data from a NASA satellite show that summertime ... than on weekends. Scientists say air pollution from humans is ... and the day of the week is evident in data ... Midweek storms tend to be stronger, drop more rain and ...
... has demonstrated the role of a gene important to ... that requires coordination of differentiation of immature neural cells ... numbers. Until now, the mechanisms regulating these distinct cellular ... will be published in the Feb. 15 issue of ...
... 2008 -- As organic farming becomes more common, ... increasingly important. In a recent study in Journal ... discrimination to determine if non-organic, synthetic fertilizers were ... the Instituto Murciano de Investigacin y Desarrollo Agrario ...
Cached Biology News:NASA data link pollution to rainy summer days in the southeast 2NASA data link pollution to rainy summer days in the southeast 3Trainor Lab characterizes gene essential for prenatal development of nervous system 2Is it organic or not? 2
... sizing single-stranded RNA ranging from 0.5 kb to 10 ... consists of nine bands in even increments (0.5 1 ... to simplify size estimation. The 1.5 kb band is ... and identification of RNA bands on the gel. All ...
Recombinant Mouse Wnt-3a...
Homo sapiens jumping translocation breakpoint...
One-step, microplate or cuvet, colorimetric, detection range 0.01 g/dL to 5 g/dL. Procedure: 5 min....
Biology Products: