Navigation Links
MHRA Issues Notice of Acceptance for Phase 3 Trial of GALNS for the Treatment of MPS IVA
Date:12/14/2010

NOVATO, Calif., Dec. 14, 2010 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has completed review of the Clinical Trial Application (CTA) and issued a notice of acceptance for the Phase 3 trial of N-acetylgalactosamine 6-sulfatase (GALNS) for the treatment of Mucopolysaccharidosis IVA (MPS IVA).  The company expects to initiate the Phase 3 pivotal trial early in the first quarter of 2011.

"This important milestone reinforces our confidence in the Phase 3 trial design for GALNS as we move another step forward in the development of a treatment option for MPS IVA," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "Results from the successful Phase 1/2 trial, along with our ongoing dialogue with health authorities in Europe and the U.S., have helped inform the design of a robust Phase 3 trial to maximize the therapy's chance for success."

The proposed Phase 3 trial would be a randomized, double-blind study with three study arms: placebo, two mg/kg every week and two mg/kg every other week in approximately 160 patients worldwide.  The primary endpoint would be 6-minute walk distance with an entry criteria of baseline walk distance no greater than 325 meters.  The company has submitted a special protocol assessment (SPA) to the FDA, with a response expected by late January.

About MPS IVThe rate of incidence of MPS IVA is as yet unconfirmed and varies among different populations but estimates vary between 1 in 200,000 live births and 1 in 250,000 live births. There are several studies that have documented the incidence as high as 1 in 76,000 live births in Northern Ireland. The estimated prevalence is between 1,000 and 1,500 patients in the U.S., EU and Japan and between 1,500 to 2,000 patients in the rest of the
'/>"/>

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

0

GOOD

Page: 1 2 3

Related medicine technology :

1. The First Annual Cervical Cancer/HPV Conference Tackles Controversial Vaccine Issues and Encourages Advocacy
2. European Unions CHMP Issues Positive Opinion on PEGINTRON(TM) Combination Therapy for Retreating Hepatitis C Patients Who Failed Previous Therapy
3. European CHMP Issues Positive Opinion for ATRIPLA(R) (efavirenz 600 mg/ emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg)
4. Bayer Issues Additional Guidance to Physicians on Trasylol(R)
5. Crystal Research Associates, LLC Issues Executive Informational Overview(R) (EIO(R)) on CEL-SCI Corporation
6. QIAGEN and Center for Molecular Medicine Create Research Collaboration for Molecular Diagnostic Markers for Breast Cancer, Other Womens Health Issues
7. Crystal Research Associates, LLC Issues Executive Informational Overview(R) (EIO(R)) on Bioniche Life Sciences Inc.
8. Lupus Under the Spotlight: U.S. Congress Shows Support for Womens Health Issues
9. European Unions CHMP Issues Positive Opinion on Sugammadex
10. Newly Published Study Shows OPKOs siRNA Bevasiranib Is Taken Up by Target Tissues in the Eye
11. Ending Cervical Cancer Featured at Summit; QIAGEN CEO Issues a Call to Global Women Leaders
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/24/2013)... 24, 2013 /PRNewswire-iReach/ -- Phoenix Houses of ... services organization, is hosting its tenth annual "Triumph For ... June 6, 2013.  More than 300 people are expected ... adolescent residential treatment centers, family therapy, and prevention programs ... accomplishments have positive influences on the health and welfare of ...
(Date:5/24/2013)... , May 24, 2013 China Biologic Products, ... leading fully integrated plasma-based biopharmaceutical company in ... recently learned from a public announcement issued by Shanghai ... company listed on Shenzhen Stock Exchange, that on May ... ("Ms. Chan"), one of the shareholders of the ...
(Date:5/24/2013)... May 24, 2013 The potential ... systemic delivery of therapeutic agents has been receiving undeniable ... delivery showing significant promise. The crucial formulation considerations for ... device play a major role in the efficiency of ... the development of new formulation techniques and devices in ...
Breaking Medicine Technology:Phoenix House Honors Allergan Chairman David Pyott And Legendary Sports Agent And Humanitarian, Leigh Steinberg 2Phoenix House Honors Allergan Chairman David Pyott And Legendary Sports Agent And Humanitarian, Leigh Steinberg 3China Biologic Comments on the Proposed Shanghai RAAS Transaction 2China Biologic Comments on the Proposed Shanghai RAAS Transaction 3China Biologic Comments on the Proposed Shanghai RAAS Transaction 4Inhalaltion Drug Delivery: 11-12 September - Berlin, Germany 2
... (AMEX: ISR ), a medical technology company ... for brain cancer, lung cancer, head and neck cancer ... and Chief Executive Officer, will co-present at RetailInvestorConferences.com. ... PM EST LINK:   www.retailinvestorconferences.com > ...
...   Leatt Corporation (OTC: LEAT.PK), a global marketer ... designed to help prevent potentially devastating injuries to the ... formation of an agreement with Whites Powersports for the ... Whites Powersports has more than 50 years experience in ...
Cached Medicine Technology:IsoRay, Inc. to Webcast, Live, at RetailInvestorConferences.com on December 1st 2Leatt Announces Distribution Agreement With Whites Powersports for New Zealand Market 2
(Date:5/25/2013)... May 25, 2013 By working diligently ... declared the integration of the 2012 Lamborghini Aventador into ... the latest entrant in the list of vehicles lined ... vehicle is all set to thrill automobile lovers in ... power and spectacular looks, the 2012 Lamborghini Aventador comes ...
(Date:5/25/2013)... According to world-renowned scholars, Queen Cleopatra ... women that ever lived. Cleopatra’s legendary beauty moved nations ... most powerful men in her time. Cleopatra was also ... her skin glowing and fresh, from her legendary milk ... oil and cyprus grass. , Cleopatra was said to ...
(Date:5/25/2013)... 25, 2013 For college students, ... week. Increasingly, they are turning to prescription stimulants ... study. For more on this, go to ... facility, warns that students who pick up Adderall, ... risk for developing a physical and psychological dependency. ...
(Date:5/25/2013)... (PRWEB) May 25, 2013 HealthCrave ... that is unique in that it doesn’t just ... focuses on treating what causes the noticeable signs ... physician Dr. James D. Weiss, M.D., the center ... aging process while helping patients to live longer, ...
(Date:5/24/2013)... A recent study by research group Harris Interactive found that ... season. Almost 138 million people are fearful of pulling on ... issues and a quarter admit they feel pressured to lose ... those anxious about their size consider the over the counter ... effort to improve diet and exercise habits ahead of peak ...
Breaking Medicine News(10 mins):Health News:Rim Fitments Provider SizeItUp Launches The Database Integration For The 2012 Lamborghini Aventador 2Health News:The Cleopatra Lift - A New Non Surgical Option to Aging Offered by Vancouver Physician 2Health News:The Cleopatra Lift - A New Non Surgical Option to Aging Offered by Vancouver Physician 3Health News:Addiction and Drug Abuse: Study Drug Adderall Abuse Spikes During Finals 2Health News:Anti-Aging Center Offers Advanced Treatment that Controls the Aging Process from Within 2Health News:PhenObestin 37.5 Can Help Make Bathing Suit Season Less Stressful 2Health News:PhenObestin 37.5 Can Help Make Bathing Suit Season Less Stressful 3
... HealthDay Reporter , MONDAY, Sept. 26 (HealthDay News) ... brain size and more problems with thinking skills as people ... who suffer from B-12 deficiencies may be greater than thought ... not be accurate, said Christine C. Tangney, lead author of ...
... study shows that 145,000 deaths could be averted in ... stronger tobacco control policies. This set of policies, as ... Organisation, consists of increasing tobacco taxes to 70% of ... public places, a complete marketing ban, well-funded tobacco control ...
... cancer patients who use a new combination of the ... Gy) before surgery have an 88 percent chance of ... randomized trial presented at the plenary session, October 3, ... Society for Radiation Oncology (ASTRO). "The results of ...
... 26, 2011 Notable levels of calcium buildup in coronary ... in people with diabetes and metabolic syndrome, according to a ... The researchers also found that individuals with diabetes or metabolic ... as low as many without these conditions. Supported by ...
... treatment for one form of albinism? Individuals ... pale skin, and light-colored irises because they have none, or ... and eyes. Affected individuals have impaired eyesight and a substantially ... to attempts to correct eyesight and counseling to promote the ...
... health challenges earlier than their fellow seniors, researchers ... lives of 8,700 older people, a Case Western ... reported physical limitations by African American women in ... finding surfaced as researchers looked generally at how ...
Cached Medicine News:Health News:Could Too Little Vitamin B-12 Shrink the Aging Brain? 2Health News:Could Too Little Vitamin B-12 Shrink the Aging Brain? 3Health News:Stronger tobacco control policy in the Netherlands would save thousands of lives 2Health News:Chemo plus radiation before surgery increases tumor response for rectal cancer 2Health News:JCI online early table of contents: September 26, 2011 2Health News:JCI online early table of contents: September 26, 2011 3Health News:JCI online early table of contents: September 26, 2011 4Health News:JCI online early table of contents: September 26, 2011 5Health News:JCI online early table of contents: September 26, 2011 6Health News:JCI online early table of contents: September 26, 2011 7Health News:African-American women develop functional challenges earlier than others 2Health News:African-American women develop functional challenges earlier than others 3
Cannula tip is specially rounded, extra smooth for safe insertion through wound and manipulation in the anterior chamber. 23G x 7/8 in (.60 x 22mm). .3mm end port. Olive shape tip is 2mm long x 1mm w...
For administering anesthetic agents into the posterior sub-Tenon's space 20G x 1in (.90 x 25mm)...
For administering local anesthetic prior to ophthalmic surgery. 27G x 1 1/4 in (.40 x 32mm). May also be used for retrobulbar administration....
... is a complete vestibular diagnostic system ... movements collected via electrodes. Its Windows-based ... use. Saccade, tracking and Slow Phase ... is facilitated by graphic comparison of ...
Medicine Products: