Navigation Links
Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)

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

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:
(Date:6/27/2016)... /PRNewswire/ - BIOREM Inc. (TSX-V: BRM) ("Biorem" or "the Company") ... shareholders, Clean Technology Fund I, LP and Clean Technology ... based venture capital funds which together hold approximately 59% ... diluted, as converted basis), that they have entered into ... holdings in Biorem to TUS Holdings Co. Ltd. ("TUS") ...
(Date:6/27/2016)... ... 27, 2016 , ... Parallel 6 , the leading software as a ... Reach Virtual Patient Encounter CONSULT module which enables both audio and video telemedicine ... team. , Using the CONSULT module, patients and physicians can schedule a face to ...
(Date:6/27/2016)...  Liquid Biotech USA , ... Sponsored Research Agreement with The University of Pennsylvania ... cancer patients.  The funding will be used to ... clinical outcomes in cancer patients undergoing a variety ... employed to support the design of a therapeutic, ...
(Date:6/24/2016)... 24, 2016 Epic Sciences unveiled a ... susceptible to PARP inhibitors by targeting homologous recombination ... The new test has already been incorporated into ... cancer types. Over 230 clinical trials ... pathways, including PARP, ATM, ATR, DNA-PK and WEE-1. ...
Breaking Biology Technology:
(Date:4/26/2016)... 27, 2016 Research and ... Biometrics Market 2016-2020"  report to their offering.  , ... The analysts forecast the global multimodal biometrics ... during the period 2016-2020.  Multimodal biometrics ... such as the healthcare, BFSI, transportation, automotive, and ...
(Date:4/14/2016)... , April 14, 2016 ... and Malware Detection, today announced the appointment of ... the new role. Goldwerger,s leadership appointment comes ... the heels of the deployment of its platform at ... behavioral biometric technology, which discerns unique cognitive and physiological ...
(Date:3/29/2016)... , March 29, 2016 ... "Company") LegacyXChange "LEGX" and SelectaDNA/CSI Protect are pleased to ... ink used in a variety of writing instruments, ensuring ... of originally created collectibles from athletes on LegacyXChange will ... analysis of the DNA. Bill Bollander ...
Breaking Biology News(10 mins):