Navigation Links
BioMarin Initiates Phase 3b Study to Evaluate the Effects of Kuvan on Neurophychiatric Symptoms in Subjects with PKU
Date:8/17/2010

NOVATO, Calif., Aug. 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the first subject has initiated treatment in a Phase 3b study (PKU-016) to evaluate the effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (PKU).  BioMarin expects to report results in the first half of 2012.

"We hope that this trial will provide quantitative data regarding the neuropsychiatric effects of Kuvan for PKU patients, and if successful, will continue to build its value proposition and to drive the continued expansion of the market," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Although we have heard anecdotal accounts from patients and physicians regarding Kuvan's impact beyond blood Phe level reduction, BioMarin has never formally evaluated these endpoints in a company trial due to the extremely short development time for Kuvan.  We are excited to have this important study underway, and if the results are positive, we plan to file for a label amendment.  We also look forward to upcoming data from several investigator-sponsored studies on the neurocognitive effects of Kuvan at the SSIEM meeting in Istanbul in late August."

The Phase 3b study is a double-blind, placebo-controlled, randomized 28-week study (2-week screening period, 13-week randomized treatment period, 13-week open-label treatment period) to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.  The study will enroll approximately 200 subjects ages twelve and above at approximately 40 sites in the U.S. and Canada.  

The primary efficacy variable for symptoms of attention deficit hyperactivity disorder (ADHD) will be evaluated using the Attention-Deficit Hyperactivity Disorder Rating Scale (ASRS) in sub
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2010 PR Newswire.
All rights reserved

Page: 1 2 3

Related medicine technology :

1. BioMarin Partner AnGes MG, Inc. Submits BLA to Japanese Ministry of Health
2. BioMarin Announces FDA Approval for Kuvan
3. BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU
4. BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease
5. BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome
6. BioMarin Initiates Phase 2 Clinical Study of PEG-PAL in PKU
7. BioMarin Acquires Huxley Pharmaceuticals, Inc.
8. BioMarin to Present at the Oppenheimer Healthcare Conference
9. BioMarin Announces Third Quarter 2009 Financial Results
10. BioMarin to Acquire LEAD Therapeutics
11. BioMarin to Present at the Credit Suisse Healthcare Conference
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/23/2015)... PHILADELPHIA , Jan. 23, 2015  Pete Rose, baseball,s all-time ... joint and arthritis pain reliever Myoflex, Ducere Pharma has announced. ... and we are excited to have him on board with ... . "He is a legendary player who is known across ...
(Date:1/23/2015)... 2015 Cord Blood America, Inc. ( www.cordblood-america.com ) (OTC ...  filed a Preliminary Proxy Statement, Schedule 14A, with the U.S. ... an update for shareholders detailing why a "YES" vote to ... Dear Shareholders, As we reflect upon the 2014 ...
(Date:1/23/2015)... January 23, 2015 Heptares Therapeutics, the clinical-stage ... announce that the Malcolm Campbell Memorial Prize for ... President of Chemistry), Fiona Marshall (Chief Scientific Officer ... and co-founder) for the seminal contributions to GPCR drug discovery ...
Breaking Medicine Technology:Pete Rose bets on Myoflex to relieve aches and pains 2Cord Blood America Announces Preliminary Proxy Filing and Details Why a "YES" Vote to Increase Authorized Shares Is Requested 2Cord Blood America Announces Preliminary Proxy Filing and Details Why a "YES" Vote to Increase Authorized Shares Is Requested 3Cord Blood America Announces Preliminary Proxy Filing and Details Why a "YES" Vote to Increase Authorized Shares Is Requested 4Heptares Scientists to Receive the Malcolm Campbell Memorial Prize for 2015 in Recognition of Their Outstanding Contribution to GPCR Drug Discovery 2Heptares Scientists to Receive the Malcolm Campbell Memorial Prize for 2015 in Recognition of Their Outstanding Contribution to GPCR Drug Discovery 3Heptares Scientists to Receive the Malcolm Campbell Memorial Prize for 2015 in Recognition of Their Outstanding Contribution to GPCR Drug Discovery 4
... was recently honored for the third year in a ... Practice Greenhealth, recognizing the organization for its outstanding environmental ... and Eastside community. As recipients of ... 15 percent of its total waste, concentrated efforts for ...
... May 30, 2012  Stiefel, a GSK (NYSE: ... entered into an agreement for the acquisition by Stiefel ... anti-inflammatory agent, WBI-1001, in all territories outside of China, ... Phase II clinical development for the treatment of psoriasis ...
Cached Medicine Technology:EvergreenHealth Wins National Award for Environmentally Responsible Healthcare for Third Year in a Row 2EvergreenHealth Wins National Award for Environmentally Responsible Healthcare for Third Year in a Row 3Stiefel, a GSK company, enters agreement with Welichem Biotech, Inc. to acquire a novel topical agent for psoriasis and atopic dermatitis 2
(Date:1/22/2015)... 2015 Autocarinsurancebest.com has released a new blog ... auto insurance policy . , Auto insurance quotes help ... is no longer necessary to call an agent or visit ... online on a single website: http://autocarinsurancebest.com/ . , Every ...
(Date:1/22/2015)... (PRWEB) January 22, 2015 Carinsurancesavings.biz has released ... accidental death and dismemberment insurance . , Purchasing an ... can be very advantageous. This type of rider provides benefits ... dismemberment can be considered a lesser form of life insurance. ...
(Date:1/22/2015)... A study recently published in ... Medicine & Science in Sports & Exercise shows ... reduce gastrointestinal (GI) problems associated with physical strain ... the double-blind, placebo-controlled study took 10 grams of ...
(Date:1/22/2015)... 23, 2015 Recently, Weddingshe.com has added many ... has announced its highest annual revenues in the past three ... popular glamorous wedding dresses. , According to the sales manager ... dress promotions for them in 2015. This point can be ...
(Date:1/22/2015)... 22, 2015 Four years since the release of ... coincided with the marriage of Avasa & Matthew Love – the ... is scheduled for release through White Swan Records on February 24, ... is a sacred path available to all of us to walk ...
Breaking Medicine News(10 mins):Health News:Commercial Auto Insurance Provides Several Important Features! 2Health News:Accidental Death and Dismemberment Insurance Can Be Purchased at Low Prices Online 2Health News:Study Shows Kyowa Hakko’s L-Citrulline May Help Reduce Gastrointestinal Injury During Strenuous Exercise 2Health News:Study Shows Kyowa Hakko’s L-Citrulline May Help Reduce Gastrointestinal Injury During Strenuous Exercise 3Health News:Weddingshe Announces A Special Offer On Its Popular Glamorous Wedding Dresses 2Health News:THE ROAD by Avasa & Matthew Love -- A New Album 2Health News:THE ROAD by Avasa & Matthew Love -- A New Album 3Health News:THE ROAD by Avasa & Matthew Love -- A New Album 4
... three of the 12 newly awarded grants from the ... prevention and treatment of prison and jail inmates. The ... are part of Seek, Test, and Treat: Addressing HIV ... initiative to date to aggressively identify and treat HIV-positive ...
... have hypertension are much more likely to have learning ... a new University of Rochester Medical Center (URMC) study ... In fact, when variables such as socio-economic levels are ... likely to have cognitive problems. "This ...
... Jenifer Goodwin HealthDay Reporter , MONDAY, Nov. 8 ... people who have never smoked may be a different disease ... genetic characteristics of lung cancer tumors in 30 people who ... The tumors of people who had never smoked had ...
... UC Davis researchers have found a new pathway for repairing ... week in the journal Proceedings of the National Academy ... greater capacity to repair oxidative damage," said Peter Beal, professor ... paper. As part of its inflammatory response, the body,s ...
... 2010) Results from a European Observational Study, ... at the Tenth International Congress on Drug Therapy ... of discontinuation and sustained virologic suppression with REYATAZ ... to five years. 1 The aim ...
... Multidetector computed tomography (CT) helps pinpoint the causes ... potentially speeding the delivery of life-saving treatments, according to ... of Radiology . "Our results suggest ... for identifying the cause of acute ischemic stroke," said ...
Cached Medicine News:Health News:Miriam receives $1.5 million from NIH to 'seek, test and treat' inmates with HIV 2Health News:Miriam receives $1.5 million from NIH to 'seek, test and treat' inmates with HIV 3Health News:Children with high blood pressure more likely to have learning disabilities 2Health News:Lung Cancer in Smokers, Nonsmokers May Be a Different Disease 2Health News:Lung Cancer in Smokers, Nonsmokers May Be a Different Disease 3Health News:New DNA repair pathway 2Health News:Study shows durable viral suppression of boosted REYATAZ in treatment-experienced HIV patients 2Health News:Using CT, radiologists can pinpoint cause of some strokes 2
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
Medicine Products: