Regulatory Focus™ > News Articles > Regulatory Recon: UniQure Hemophilia Gene Therapy Shows Promise, India Readies Biosimilar Guidelines

Regulatory Recon: UniQure Hemophilia Gene Therapy Shows Promise, India Readies Biosimilar Guidelines (7 January 2016)

Posted 07 January 2016 | By Michael Mezher 

Regulatory Recon: UniQure Hemophilia Gene Therapy Shows Promise, India Readies Biosimilar Guidelines (7 January 2016)

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

  • UniQure's Gene Therapy Boosts Clotting in Two Hemophiliacs (Bloomberg) (Reuters) (Press)
  • House Votes to Send Bill to Repeal Health Law to Obama's Desk (NYTimes)
  • FDA 2015: A Look Back (and Ahead) – Part 2: Medical Product Safety and Oversight (FDA Voice)
  • New Data Could Boost 'Switching' To Biosimilar Infliximab (SCRIP-$) (MNT)
  • We Don't Just Need Precision Medicine, We Need Precision Health (Forbes)

In Focus: International

  • Ebola Treatment Using Plasma From Survivors Is Not Effective, Study Says (NYTimes) (Reuters)
  • Patient Access, Cost Inhibit Adoption of Biosimilars in Poorer Global Markets (Thomson Reuters)
  • EU To Test Merging & Splitting Of Licences Under Mutual Recognition/Decentralized Procedures (SCRIP-$)
  • June 17 Likely Date for Adoption of New EU Regs; Regulators Feel The Strain (Clinica-$)
  • India's Revised Biosimilar Guidelines Ready In A Month (PharmAsiaNews-$)

US: Pharmaceuticals and Biotechnology

  • Does cancer screening saves lives? Unclear, researchers say (Reuters) (MNT)
  • A lesson in DTC: What happened when a Vogue contributor took Addyi for a test run (BioPharmaDive)
  • "Temporary Compounding" To Mitigate Off-Patent Monopolies (RPM Report-$) (Focus)
  • Study Finds Birth Control Pill Use Isn't Associated With Birth Defects (NPR)
  • US biosimilars in 2016: Where we're at following Zarxio's breakthrough (BioPharma-Reporter)
  • Pharma Companies Focus Pre-Clinical New Product Planning On Medical Affairs Expenditures (Cutting Edge Information)
  • CDER Compliance Office Director Must Recuse Herself From Most Duties (Pink Sheet-$)
  • Pharma Needs A Leader In 2016 (Forbes)
  • Will CROs adopt remote patient monitoring in 2016? (Outsourcing-Pharma)
  • Regeneron's Stock Just Got Slammed By A Side Effect That Wasn't (Forbes)
  • Native advertising for pharma: Winning format or slippery slope to regulatory headaches? (FiercePharmaMarketing)
  • FDA sends fewer drug promotion action letters in 2015--but is that a good thing for pharma? (FiercePharmaMarketing)

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

  • FDA Grants Priority Review to AbbVie for Supplemental New Drug Application for VIEKIRA PAK (Press)
  • Vyome Biosciences Announces FDA Acceptance of Investigational New Drug Application in the US for its Lead product VB-1953 to Treat Moderate-to-Severe Acne (Press)
  • Pharming completes patient enrolment in Phase 2 Ruconest trial (EPR)
  • Anacor Pharmaceuticals Submits New Drug Application to the FDA for Crisaborole Topical Ointment, 2% for the Treatment of Mild-to-Moderate Atopic Dermatitis (Press)
  • Glaukos Will Begin Phase II Clinical Trial for iDose Travoprost Intraocular Implant in Glaucoma Patients (Press)
  • Aeglea BioTherapeutics Announces FDA Acceptance of Its Investigational New Drug Application for AEB1102 for the Treatment of Arginase I Deficiency (Press)
  • Innocrin Pharmaceuticals, Inc. Granted Fast Track Designation by FDA for VT-464 Treatment of Patients with Metastatic Castrate-resistant Prostate Cancer (Press)
  • Transgene announces first patient randomized in multinational Phase 3 trial for Pexa-Vec oncolytic immunotherapy in advanced liver cancer (MNT)
  • PROGNOSIS Study Published in The New England Journal of Medicine Reveals Innovative Roche Blood Test Can Be Used as a Predictive Tool for Preeclampsia (Press)
  • Important Update Regarding CIRM's Partnered Clinical Trial for Patients with Metastatic Melanoma (CIRM)

US: Medical Devices

  • A volunteer group out of the University of Minnesota is looking to increase pediatric medical device innovation (MedCityNews)
  • FDA warns Sorin on Stockert heater/cooler device (MassDevice) (Focus)
  • SRS Medical Receives FDA IDE Approval for Expanded Indications Study of Spanner Prostate Stent (Press)
  • Pathway Genomics Debuts First Genomic Wellness App Powered by IBM Watson (IBM)
  • Medtech wins FDA nod for ROSA spine robot (MassDevice)
  • MST Receives FDA Clearance for "Follow Me" Image-Guided Software for Minimally Invasive Robotic Surgery (Press)
  • NeuroMetrix Announces FDA 510(k) Clearance of Next Generation Quell Wearable Pain Relief Technology at the 2016 Consumer Electronics Show (Press)

US: Assorted and Government

  • GSK Faces Whistleblower Suit Claiming Study Mistakes (Law360-$)
  • Indiana bill encourages sale of meth-resistant PSE products (DSN)
  • FDA Broadens Arsenal in Fight Against Kratom (FDA Law Blog)
  • US Marshals seize dietary supplements containing kratom (FDA)
  • DEA investigates painkiller epidemic in West Virginia (CBS)
  • This Time May Be Different: Today's Biotech Market In Context (LifeSciVC)

Upcoming Meetings and Events              


  • Germany Up Close: BfArM Prepares For A Wave Of App Activity (Clinica-$)
  • Partnership between NHS and Roche targets rare heart condition (Pharmaceutical Journal-$)
  • Novo Nordisk files for regulatory approval of long-acting factor IX in the EU for the treatment of haemophilia B (Press)
  • EDMA and Eucomed Adopt Code of Business Practice Brings Changes to Medical Congresses (Policy and Medicine)


  • WHO to set up centre of excellence in regulatory science at FDCA Gujarat soon (PharmaBiz)
  • Telangana drug inspectorsget training from USFDA (The Hindu)
  • SUGAM, CDSCO'S Online Portal For Import Applications, Now Requires Pre-Registration (24 Insight)
  • India needs to build a safety net around consumers before online pharmacies are permitted: Dr Jagashetty (PharmaBiz)



  • Japanese drugmaker plans companion diagnostic for Opdivo (Nikkei)

Other International

  • Shimadzu Global Pharma Summit gathers veterans to share regulatory challenges (BioSpectrum)
  • Brazilian Government Owes Pharma R$1bn (SCRIP-$)

General Health and Other Interesting Articles

  • Patients leaving hospitals often don't understand care plans (Reuters)

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.

Categories: Recon, Regulatory News

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