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:6/23/2016)... Houston Methodist Willowbrook Hospital has signed ... to serve as their official health care provider. ... will provide sponsorship support, athletic training services, and ... volunteers, athletes and families. "We are ... and to bring Houston Methodist quality services and ...
(Date:6/23/2016)... ... June 23, 2016 , ... Supplyframe, the Industry Network ... Supplyframe Design Lab . Located in Pasadena, Calif., the Design Lab’s mission is ... projects are designed, built and brought to market. , The Design Lab is ...
(Date:6/23/2016)... 23, 2016 Andrew ... http://doi.org/10.17925/OHR.2016.12.01.22 Published recently in ... journal from touchONCOLOGY, Andrew D Zelenetz , ... cancer care is placing an increasing burden on ... biologic therapies. With the patents on many biologics ...
(Date:6/23/2016)... 22, 2016  Amgen (NASDAQ: AMGN ) ... QB3@953 life sciences incubator to accelerate the development ... laboratory space at QB3@953 was created to help high-potential ... for many early stage organizations - access to laboratory ... Amgen launched two "Amgen Golden Ticket" awards, providing each ...
Breaking Biology Technology:
(Date:6/22/2016)... , June 22, 2016   ... management and verification solutions, has partnered with ... software solutions for Visitor Management, Self-Service Kiosks ... provides products that add functional enhancements to ... provides corporations and venues with an automated ...
(Date:6/15/2016)... 15, 2016 Transparency Market ... Recognition Market by Application Market - Global Industry Analysis Size ... to the report, the  global gesture recognition market ... and is estimated to grow at a CAGR ... 2024.  Increasing application of gesture recognition ...
(Date:6/2/2016)... 2016 The Department of Transport Management ... 44 million US Dollar project, for the , ... Personalization, Enrolment, and IT Infrastructure , to ... and implementation of Identity Management Solutions. Numerous renowned international vendors ... Decatur was selected for the most compliant and ...
Breaking Biology News(10 mins):