FDA News, Week of September 7, 2015 – Drug and Device Digest

FDA Voice

FDA Taking Genomic Testing to the Next Level


  • Based on President Obama’s Precision Medicine Initiative (PMI)
  • New regulatory strategies for Next Generation Sequencing (NGS)  based clinical test – analyzing and interpreting a  unique genetic makeup and alterations that may impact health
  • December 2014 : Preliminary Discussion paper
  • November 12th ; Public Workshop – Analytical performance evaluation standards
  • November 13th : Public Workshop –  Current challenges in clinical validation of NGS tests

OpenFDA Makes Medical Device-Related Data Easier to Access and Use


  • Releasing information on medical devices for easy access to researchers, scientists, and developers
  •  6,000 device classification records
  • 24,000 registrations of device companies and establishments
  • >100,000 devices
  • 30,000 PMAs and supplements
  • 141,000 510(k)s and  de novo requests
  • 9,500 device recalls
  • 4.2 million adverse event reports
  • Safeguards: Protect personal identity and confidential commercial information
  • Active in GitHub and StackExchange, encourage external parties to participate

SUMMARY  : FDA initiatives to  spur innovation, advance scientific research, engage public and foster collaboration

FDA approved

XURIDEN® (uridine triacetate, Wellstat Therapeutics Corporation, Gaithersburg, MD)

Rare disease treatment for hereditary orotic aciduria , a rare metabolic disorder, reported in approximately 20 patients worldwide. Patients cannot synthesize adequate amounts of uridine and consequently can suffer from hematologic abnormalities, failure to thrive, a range of developmental delays, and episodes of crystalluria leading to obstructive uropathy

Reg Pathway : Orphan designation, priority review, granted rare pediatric disease priority review voucher

Indication : treatment of hereditary orotic aciduria

Efficacy :

Open-label study in 4 patients (3 to 19 years), 6 weeks.

  • Primary endpoint was stability of hematological paramters (eg neutrophil count, percent neutrophils, white blood cell count, mean corpuscular volume)
  • Patients 1 and 3 met pre-specified criteria
  • Patient 2 pre-specified criteria remained stable
  • Patient 4 did not meet the pre-specified endpoint
  • In 6-mo extension treatment, hematologic parameters for patients normalized or remained stable
  • Growth improved or remained stable

Safety : No adverse reactions

SUMMARY  Congratulations to Wellstat Therapeutics for pursuing and succeeding in drug development in an ultra rare potentially life-threatening, genetic disorder

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