Regulatory Focus™ > News Articles > Regulatory Recon: UniQure Drops Quest for FDA Approval of its Gene Therapy & A Second Patient Dies i

Regulatory Recon: UniQure Drops Quest for FDA Approval of its Gene Therapy & A Second Patient Dies in Beloranib Trial (2 December 2015)

Posted 02 December 2015 | By Michael Mezher 

Regulatory Recon: UniQure Drops Quest for FDA Approval of its Gene Therapy & A Second Patient Dies in Beloranib Trial (2 December 2015)

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing. Want to read Recon as soon as it's posted? Follow @Michael_Mezher, @Zachary Brennan and @RAPSorg on Twitter.

In Focus: US

In Focus: International

  • Singapore HSA Revises Medical Device Change Notification System (Emergo)
  • Compensation System for Vaccine Injuries Needed for Diseases Such as Ebola, Policy Experts Say (ICT) (PLoS)
  • India restores federal funding for AIDS program after criticism (Reuters)
  • First Japan-based Apple ResearchKit study, Heart and Brain, launches (MobiHealthNews)

US: Pharmaceuticals and Biotechnology

  • Gene Drive Research Takes Aim at Malaria (NIH)
  • US Still in Danger of Losing War on AIDS, C.D.C. Director Says (NYTimes)
  • Adaptive Designs Cut Costs and Improve Efficiency (Geeks Talk Clinical)
  • Female Viagra: Discrimination or Medicalization or Something Else? (Harvard Bill of Health)
  • Fruit-flavored drugs to cut child TB deaths, combat 'superbugs' (Reuters)
  • Anti-HIV pill, taken as needed, prevents infection in gay men (Reuters) (Washington Post)

US: Pharmaceuticals and Biotechnology: Clinical Study Results, Filings and Designations

  • Cellceutix' Kevetrin gets Rare Pediatric Disease designation from FDA (PharmaLetter-$)
  • PhaseBio Receives FDA Orphan Drug Designation for Vasomera (PB1046) for the Treatment of Cardiomyopathy Associated With Dystrophinopathies (Press)
  • Positive Phase II For Sanofi-Chasing Valneva/GSK C.Diff Vaccine (SCRIP-$)
  • MabVax Therapeutics Files IND for Phase I Clinical Trial with HuMab 5B1 as a Therapeutic for Pancreatic Cancer (Press)
  • Enanta Pharmaceuticals Announces FDA Acceptance of AbbVie's New Drug Application for a Once-Daily Formulation of VIEKIRA PAK (Press)
  • Preliminary Phase 2 Data Demonstrate Tumor Treating Fields is Safe in Patients with Brain Metastases Originating from Non-Small Cell Lung Cancer (Press)
  • MedDay's MD1003 study fails to reach primary endpoint (PharmaLetter-$)
  • Newron Initiates U.S. Phase II Trial in Patients with Schizophrenia (Press)
  • Advaxis Receives Orphan Drug Designation in the European Union for ADXS-HER2 for the Treatment of Osteosarcoma (Press)
  • HIV: ABIVAX reports healthy volunteers Phase I data for ABX 464 (MNT)
  • Lexicon's telotristat etiprate achieves positive top-line results in second Phase III trial (PharmaLetter-$)
  • New Data Show Lilly's Once-Weekly Trulicity (dulaglutide) is Effective as Add-on Treatment to Sulfonylurea (Press)

US: Medical Devices

  • Diagnosing malaria with a cell phone (Reuters)
  • Philips announces expanded indications for its SkyFlow X-ray imaging software at RSNA 2015 (Press)
  • A Funky Preemption Decision from California (Drug and Device Law)
  • Alcresta Pharmaceuticals Receives FDA Clearance for RELiZORB (Press)
  • FDA Grants StimRelieve IDE Approval For A Wireless CranioFacial Nerve Stimulator (CFNS) For The Treatment of CranioFacial Neuropathic Pain (Press)
  • Guided Therapeutics and FDA Review Team Agree on Path Forward for LuViva Advanced Cervical Scan PMA Application (Press)
  • Important step toward preventing and treating some MRSA post-implant infections (MNT)
  • Greatbatch wins FDA PMA for Algovita SCS (MassDevice)
  • PAD: Boston Scientific wins FDA, CE Mark nods for thrombectomy catheter (MassDevice)
  • EDDA Receives FDA Clearance for IQQA-Guide Intra-operative 3D Navigation System (medGadget)

US: Assorted and Government

  • Has DC Crossed the Rubicon in Curbing Drug Prices? (Morning Consult)
  • Drug Prices are too Damn High. Here's How to Fix Them (Wired)
  • Defending 'Salt Patents' In Hatch-Waxman Cases (Law360-$)
  • Califf Nomination Gets Fishy (Pink Sheet-$)

Upcoming Meetings and Events              


  • NICE extends treatment options for chronic hepatitis C (Pharmaceutical Journal-$)
  • ABPI response to new NICE guidance on the management of type 2 diabetes (ABPI)
  • All Device Notified Bodies Need Redesignating Soon: Commission Urgently Needs To Outline Procedures (SCRIP-$)
  • Pfizer lung cancer treatment gets expanded EU approval (Pharmafile)
  • Janssen Diagnostics Garners CE-IVD Mark for Flu/RSV Panel on Biocartis Platform (GenomeWeb)
  • Good But Could Be Better: Industry On EMA Proposals For Improving Early Access Schemes (SCRIP-$)
  • Puma preps its once-vaunted cancer drug for European submission (FierceBiotech) (Press)
  • Early phase clinical research: is Russia the next big market player? (PharmaLetter-$)
  • HiberGene Receives CE Mark for Meningitis MDx (GenomeWeb)
  • Basilea receives EU market nod for isavuconazole to treat invasive aspergillosis and mucormycosis (PharmaLetter-$)
  • Horizon Pharma plc receives European Commission approval for RAVICTIi oral liquid for the treatment of urea cycle disorder (MNT)


  • Aurobindo gets USFDA nod for multi disease treatment injection (Economic Times)
  • Wockhardt's Chikalthana plant gets GMP certificate from UK MHRA (Economic Times)
  • Over 3000 adverse transfusion reaction reports generated till date through reporting system of HvPI (PharmaBiz)
  • NPPA's Pharma Jan Samadhan scheme gets good response, gets 295 complaints since launch in March (PharmaBiz)


  • Japan recognizes Cyberdyne's robotic suit as medical device, widespread use anticipated (Japan Times)

Other International

  • Merck and Uganda Ministry of Health Partner to Fight Diabetes and Cancer (PharmaAfrica)

General Health & Interesting Articles

  • Ethicists square off over editing genes in human embryos (Reuters)
  • Fewer People Are Getting Diabetes, But The Epidemic Isn't Over (NPR) (Washington Post)
  • HHS: Dramatic Downturn in Hospital-Acquired Conditions (MedpageToday)
  • Wales switches to 'opt-out' system of organ donation (EurActiv)
  • Discovery Explains Why We Do Not Constantly Become Ill Despite Viruses and Bacteria (ICT)

Regulatory Reconnaissance is our daily intelligence briefing for the regulatory affairs space, bringing you the top regulatory news stories from around the globe. Each weekday morning, we aim to bring you the latest highlights of new approvals, meetings, legal and political developments, regulations and guidance, and the latest trends with the potential to impact regulatory affairs professionals and the industry in which they work.

Need to contact the editor of Regulatory Reconnaissance? Find him on Twitter at @Michael_Mezher or send him an email at

A story's inclusion in Regulatory Reconnaissance does not imply endorsement by Regulatory Focus or RAPS.

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