Wellstat Therapeutics Receives “Rare Impact Award” From the National Organization for Rare Disorders

GAITHERSBURG, Md.--()--Wellstat Therapeutics Corporation announced that the National Organization for Rare Disorders (NORD) has honored the company with a 2016 Industry Innovation Award as part of the organization’s 2016 Rare Impact Awards program. David M. Wohlstadter accepted the award on behalf of Wellstat at NORD’s Rare Impact Awards ceremony at the Warner Theatre in Washington, D.C. on May 17.

Wellstat received an Industry Innovation Award for the development of XURIDEN™ and the positive impact the company has made on patients’ lives by providing the first approved treatment for patients with hereditary orotic aciduria (HOA).

XURIDEN received FDA approval on September 4, 2015 for the treatment of HOA, a rare, potentially life-threatening genetic disorder in which patients cannot synthesize adequate amounts of uridine. This can result in hematologic abnormalities, failure to thrive, a range of developmental delays, and obstructive uropathy due to crystalluria. Untreated, severe cases can be fatal.

“We are honored and humbled to receive a 2016 Rare Impact Award,” said David M. Wohlstadter. “Developing novel therapeutics, particularly for patients with rare disorders that are often overlooked due to small patient populations, is a challenging and rewarding undertaking. We thank NORD for shining a light on the good work that has been done -- and still remains to be done -- for patients struggling with rare diseases.”


XURIDEN is a pyrimidine analog for uridine replacement indicated for the treatment of hereditary orotic aciduria.

Important Safety Information

• Contraindications – None

• Warning and Precautions – None

• Adverse reactions – No adverse reactions were reported with XURIDEN in patients with HOA.

• Pregnancy – There are no available data on XURIDEN use in pregnant women to inform drug-associated risk.

• Lactation – There are no data on the presence of XURIDEN in human milk, the effect on the breast-fed infant or the effect on milk production. The development and health benefits of breastfeeding should be considered along with the mother’s clinical need for XURIDEN and any potential adverse effects on the breastfed infant from XURIDEN or from the underlying maternal condition.

• Pediatric – The safety and effectiveness of XURIDEN have been established in pediatric patients.

For the full Prescribing Information go to http://www.xuriden.com/FPI.pdf.

About HOA

Hereditary orotic aciduria (HOA) is a genetic disorder caused by mutations reducing the activity of uridine monophosphate synthase (UMPS), which converts orotic acid to UMP. Uridine deficiency underlies many of the symptoms of HOA, including hematologic abnormalities, developmental delays, failure to thrive, and if untreated, early mortality. Orotic acid biosynthesis is normally subject to feedback inhibition by intracellular uridine nucleotides and is therefore overproduced in HOA. The resulting increased levels of orotic acid can cause crystalluria and occasional obstructive uropathy.

The goal of uridine replacement therapy in HOA is to provide the uridine that these patients cannot make adequately themselves in order to restore intracellular uridine nucleotide concentrations, enabling and supporting the biosynthetic functions of pyrimidine nucleotides and their derivatives, and also normalizing orotic acid production.

About Wellstat Therapeutics

Wellstat Therapeutics Corporation is a privately-held biopharmaceutical company located in Gaithersburg, Maryland. Wellstat Therapeutics is committed to discovering, developing and commercializing products that will provide new and improved treatments for patients in the fields of oncology and metabolic, neurometabolic and neurodegenerative diseases. Wellstat Therapeutics is part of the Wellstat group of companies, http://www.wellstat.com. For more information, please visit the website at http://www.wellstattherapeutics.com.


Torch Communications
Jeremiah Hall, 415-349-5016


Torch Communications
Jeremiah Hall, 415-349-5016