Navigation Links
Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)
Date:2/28/2012

NOVATO, Calif., Feb. 28, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7.  MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS).  UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7.  MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University.  su

"Obtaining orphan drug designation for UX003 is a significant achievement for Ultragenyx that adds value to our development pipeline and attests to the importance of this product candidate in filling an unmet medical need," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We look forward to continuing to collaborate with Dr. Sly and colleagues at St. Louis University to advance this urgently needed therapy into clinical testing." 

The Orphan Drug Designation program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.  Among the benefits of orphan designation in the US are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received.

About MPS 7

Mucopolysaccharidosis type 7 (MPS 7), also known as Sly syndrome, is a rare genetic, metabolic disorder and is one of 40 different lysosomal storage disorders.  MPS 7 is caused by the deficiency of Beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulfate and heparan sulfate.  These complex GAG carbohydrates are a critical component of many tissues. The inability to properly breakdown GAGs leads to a progressive accumulation in many tissues and multi-system disease.  There are a wide variety of clinical symptoms including enlarged organs, stiff joints, respiratory disease and cardiac complications.

While its clinical manifestations are similar to MPS 1 and MPS 2, MPS 7 is one of the rarest among the MPS disorders and is likely underdiagnosed as are most rare diseases. MPS 7 has a wide spectrum of clinical manifestations and can present as early as at birth or in older patients with less rapidly progressive disease. There are no approved therapies for MPS 7 today.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market.  The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases.  Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for GNE myopathy, also known as hereditary inclusion body myopathy (HIBM).  UX001 program has been granted orphan drug designation in the U.S.

The company is led by an experienced management team in rare disease therapeutics.  Ultragenyx is striving toward an improved model for successful rare disease drug development which has the potential to increase efficiency while maintaining appropriate safety and efficacy standards. The company believes that it can deliver significant value to patients by building a diverse and high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.

For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.


'/>"/>
SOURCE Ultragenyx
Copyright©2010 PR Newswire.
All rights reserved

Related biology technology :

1. NanoLogix Instructs Third-Party to Prepare US FDA Submittals and is Granted US and Chinese Patents
2. BioDrain Medical, Inc. Granted Canadian Patent for its Streamway® Surgical Fluid Management System
3. Cephalon Granted Temporary Restraining Order in AMRIX® Patent Litigation
4. Ganeden Biotech Granted 100th Probiotic Patent
5. Biomerix Granted US Patent for Its Proprietary Biomaterial Platform Technology
6. EmergingMed Granted Patent
7. Spectral granted IDE approval by U.S. FDA to conduct pivotal trial for Toraymyxin(TM)
8. Appeal of Lawsuit by Former Officer Dismissed; Request for Sanctions Granted
9. YM BioSciences granted two US patents for AeroLEF(R)
10. Viron granted U.S. Patents for Organ Transplant and Arthritis Drug Candidates
11. Hahnemann University Hospital Granted Magnet(R) Status
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/18/2017)... ... August 18, 2017 , ... ... will feature Federal Hybrids, Inc. in an upcoming episode, scheduled to broadcast fourth ... Farmer will explore Federal Hybrids, the independent, family-owned seed company. Educating audiences about ...
(Date:8/17/2017)... , ... August 17, 2017 , ... ... stock market news outlet had provided a research update on Aytu Bioscience and ... TRT product. , According to Soulstring, prescription rates for Natesto® have more than ...
(Date:8/16/2017)... King of Prussia, PA (PRWEB) , ... August 16, 2017 , ... ... leaders will be taking part in sessions at the ISPE Annual Meeting and ... the Marriott Marquis San Diego Marina. The event’s theme is “Driving innovation to advance ...
(Date:8/16/2017)... ... August 16, 2017 , ... ... a different cell type. Many treatments for specific cancers, such as breast, prostate, ... targeted treatment is androgen deprivation therapy for advanced prostate cancer. , This ...
Breaking Biology Technology:
(Date:3/30/2017)... 30, 2017 Trends, opportunities and forecast in ... by technology (fingerprint, AFIS, iris recognition, facial recognition, hand ... by end use industry (government and law enforcement, commercial ... banking, and others), and by region ( North ... Asia Pacific , and the Rest of ...
(Date:3/27/2017)... 27, 2017  Catholic Health Services (CHS) has ... Society (HIMSS) Analytics for achieving Stage 6 on ... . In addition, CHS previously earned a place ... an electronic medical record (EMR). "HIMSS ... of EMR usage in an outpatient setting.  This ...
(Date:3/23/2017)... report "Gesture Recognition and Touchless Sensing Market by Technology (Touch-based and Touchless), Product ... by MarketsandMarkets, the market is expected to be worth USD 18.98 billion by ... Continue Reading ... ...      (Logo: http://photos.prnewswire.com/prnh/20160303/792302) ...
Breaking Biology News(10 mins):