Navigation Links
BioMarin Initiates Pivotal Phase 3 Trial for GALNS for the Treatment of MPS IVA
Date:2/1/2011

NOVATO, Calif., Feb. 1, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.  

"In under two years, we have progressed the GALNS program from Clinical Trial Application to initiation of the Phase 3 trial.  We have received FDA feedback and have finalized the design of the Phase 3 pivotal trial," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "The study will be conducted at approximately 40 centers worldwide including Brazil, Japan, Taiwan, most Western European countries, Canada and the U.S.  The trial is expected to enroll approximately 160 subjects and will be the largest enzyme replacement therapy trial conducted. There are no therapeutic options for MPS IVA patients who have a high unmet medical need. Initiation of this well-designed pivotal study is an important milestone for both the company and the MPS IVA community."

The Phase 3 trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of GALNS in patients with MPS IVA.  The study will explore doses of two mg/kg/week and two mg/kg/every other week for a treatment period of 24 weeks.  The primary endpoint is the six-minute walk test, and the secondary endpoints are the three-minute stair climb test and urine keratan sulfate concentration.  

About MPS IVMucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the thorax impairs respiratory function, and odontoid hypoplasia and ligamentous laxity cause cervical spinal instability and potential cord compression. Other symptoms may include hearing loss, corneal clouding, and heart valvular disease. Initial symptoms often become evident in the first five years of life.  Depending on severity of the disease, age of diagnosis will vary.

The 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 world for a total of 2,500 to 3,500 patients. Over 1,000 MPS IVA patients worldwide have been identified through The International Morquio Organization (IMO) survey and the BioMarin MorCAP registry program.

About BioMarinBioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Other product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, and BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking StatementThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the development of its program for MPS IVA, and particularly the timing and conduct of clinical trials related thereto, expectations regarding other clinical and preclinical programs, and the potential market for GALNS, if approved. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results of current and planned pre-clinical trials related to BioMarin's development programs and particularly the enzyme replacement therapy for MPS IVA; the content and timing of decisions by the U.S. Food and Drug Administration, EMEA and other regulatory agencies, particularly with respect to the enzyme replacement therapy for MPS IVA, the actual number of MPS IVA patients in the developed world; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2009 Annual Report on Form 10-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, Naglazyme® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.

Firdapse™ is a trademark of BioMarin Huxley Ltd.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.Contact:Investors

MediaEugenia Shen

Bob PurcellBioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.(415) 506-6570

(415) 506-3267
'/>"/>

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

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:2/12/2016)... Ambulances announces its first delivery in the state of ... Emergency Medical Services (EMS) consisting of four vehicles: three ... Emergency Vehicles in Lecanto, FL , USA, ... the latest in Demers, ongoing expansion of sales.  ... --> Benoit LaFortune , Executive Vice President at Demers. ...
(Date:2/12/2016)...  Memorial Hermann Health System has teamed up with ... bring a one-of-a-kind experience to pediatric patients at ... as 360-degree video and Google Cardboard, Howard was able ... giving the patients and their families an unexpected, and ... on video . Memorial Hermann IRONMAN ...
(Date:2/12/2016)... Ontario , Feb. 12, 2016  Aralez Pharmaceuticals ... pharmaceutical company, today announced the Company will ring the Nasdaq ... New York at 4:00 p.m. ... formation of Aralez. Adrian Adams , ... be held 3:50 to 4:00 p.m. ET.  A live ...
Breaking Medicine Technology:
(Date:2/12/2016)... ... February 12, 2016 , ... For Coast Dental dentist Everet Lake, ... and dental assistant Terrell Moore shortly before 7 a.m. to volunteer at Friday’s Dentistry ... donating their time and skills to help hundreds of uninsured and underinsured people receive ...
(Date:2/12/2016)... ... February 12, 2016 , ... Nearly every health website ... health. These articles generally list between five and 15 foods that should be ... every one of these lists and believes that nutritious eye healthy foods are ...
(Date:2/12/2016)... ... February 12, 2016 , ... US Sport Camps is pleased to announce ... located in Norwalk, serves as the host site and directing the camps is PGA ... have had successful camps in recent years around Des Moines and are fortunate to ...
(Date:2/12/2016)... ... February 12, 2016 , ... The Central Coast Autism Spectrum Center (CCASC) invites teens ... provide a night of fun for teens with and without special needs to gather in ... included at the event. The dance will take place on Saturday, Feb. 13 from 7 ...
(Date:2/12/2016)... Mi (PRWEB) , ... February 12, 2016 , ... Basketball ... unique and integral part of the Peety PoppersTM series, sign language translation is featured ... message of health and wellness in Peety PoppersTM lessons has a sign language translator ...
Breaking Medicine News(10 mins):