Regulatory Focus™ > News Articles > Regulatory Recon: EMA Approves First Gene Therapy in Children, New Study Validates Statin Intoleranc

Regulatory Recon: EMA Approves First Gene Therapy in Children, New Study Validates Statin Intolerance in Some Patients (4 April 2016)

Posted 04 April 2016 | By Michael Mezher 

Regulatory Recon: EMA Approves First Gene Therapy in Children, New Study Validates Statin Intolerance in Some Patients (4 April 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

In Focus: International

  • Sanofi to launch dengue mass vaccination programme (Financial Times) (BioSpectrum)
  • EU Recommendations for Composition of 2016/2017 Seasonal Influenza Vaccines (EMA)
  • Samsung Brings in the Lawyers for Biosimilars Push (Reuters) (WSJ-$) (Bio-PharmaReporter)
  • A New Decree on Fees Chargeable by the Finnish Medicines Agency Takes Effect on 1 April 2016, Reducing Some of the Fees (FIMEA)
  • Europe Gives Green Light to First Gene Therapy for Children (Reuters) (WSJ) (EMA)
  • Medtech Regulation in Latin America: Why Harmonization Matters (Clinica-$)

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US: Pharmaceuticals & Biotechnology

  • An Open Letter To The FDA, RE: Please Fix The National Drug Code Soon(RxTrace)
  • Podcast: How Wall Street reacts when a patient dies in a clinical trial (STAT)
  • A Valeant Boo-Boo May Portend Bigger Errors (NYTimes)
  • Drugmakers race to prescribe preventive migraine remedies (Financial Times)
  • How I learned to overcome my bias against for-profit medicine (STAT)
  • The Other Opioid Crisis (DD&D)
  • Viewing Recent Opioid Regulations In Context (Law360-$)
  • Biosimilar Developments Continue at a Rapid Pace (National Law Review)
  • Whitepaper: Bioanalytical support in the development of biologics (EPR)
  • Whitepaper: Simultaneous in situ real-time monitoring of a bioprocess (EPR)
  • Payer Briefs: Express Scripts Pricing Pilot; HHS Risk Adjustment; Value Frameworks (Pink Sheet-$)
  • FDA's Improved Forensics Uncovers More Manufacturing Data Integrity Issues (Pink Sheet-$)
  • Nuplazid's Black Box: Warning May Depend On Antipsychotic Classification (Pink Sheet-$)
  • Right-to-die advocates create cheaper lethal drug mix after company doubles price (The Guardian)
  • FDA Proposes Illuminating OTC Monograph Process With Sunscreen TEA Framework (Tan Sheet-$)

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

  • Effect of Losmapimod on Cardiovascular Outcomes in Patients Hospitalized With Acute Myocardial Infarction (JAMA)
  • Interim Phase III Data Show Praxbind (idarucizumab) Reverses Dabigatran (Pradaxa) in Emergency Situations (Press)
  • Chugai Pharma Europe Announces Positive Phase III Results of Aplidin (Press)
  • Hansa Medical: FDA Clears Hansa Medical's IND Application for IdeS in Kidney Transplantation (Press)
  • First Approval by FDA for Contrast Enhanced Ultrasonography of the Liver Received by Bracco Diagnostics Inc. for LUMASON (Press)

US: Medical Devices

  • Food and Drug Administration Modernization Act of 1997: Modifications to the List of Recognized Standards, Recognition List Number: 041 (FDA)
  • Class 1 Device Recall Simplexa HSV 1 & 2 Direct and Simplexa Group A Strep Direct (FDA 1, 2)
  • When Data Meets Genomics, Where Does Computing Power Come From? (Xconomy)
  • Philips enrolls 5,000+ in 3 trials of Volcano's blood pressure measurement technology (Fierce)
  • Navidea Updates Clinical Development Plan for Diagnostic Use of Lymphoseek IV in Rheumatoid Arthritis (Press)
  • St. Jude Medical CardioMEMS HF System Prompts Changes That Improve Heart Failure Management and Reduce Hospitalizations (Press)

US: Assorted & Government

  • The Growing Mess of the Supreme Court's Contraception Case (NYTimes)
  • FDA proposes limit on inorganic arsenic in infant rice cereal (Reuters)
  • The Long Swim Continues As Groups Sue FDA Over GE Salmon (FDA Law Blog)
  • Inter Partes Reviews Just Got More Expensive (SCRIP-$)

Upcoming Meetings & Events


  • Seven new therapies jump towards EU approval (PharmaTimes)
  • EU recommends conditional approval of J&J's blood cancer drug (Reuters)
  • Amicus's Fabry disease drug gets positive EU recommendation (Reuters) (WSJ-$) (Xconomy)
  • Updated PIM designation statistics (MHRA)
  • Bristol-Myers Squibb Receives Positive CHMP Opinion for Opdivo (nivolumab) in Combination with Yervoy (ipilimumab) for Treatment of Advanced Melanoma (Press)
  • Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 29 March - 1 April 2016 (EMA)
  • Added benefit of Edurant not proven, says IQWiG (PharmaLetter-$)
  • BioLineRx's Novel Treatment for Non-Surgical Removal of Skin Lesions Receives CE Mark Approval (Press)
  • Medtronic Announces Early Data and Completes Enrollment in CE Mark Cohort in Drug-Filled Stent Trial (Press)


  • Daiichi Sankyo announces closure of its Hiratsuka Plant (BioSpectrum)


  • NHRC seeks report from government on its stand on generic medicines (Economic Times)
  • India Chapter Of USTR 2016 NTW Focuses On IPR, Import Licenses And Tariffs (24 Insight)
  • Leading 15 global pharma companies post dismal performance in 2015 (PharmaBiz)
  • TN Drug Dept initiates regular inspections of retail shops to ensure no sale of banned drugs (PharmaBiz)
  • Health ministry to stop trivalent OPV, switch to bivalent polio vaccination from April 25 (PharmaBiz)
  • Latest Indian Price Caps Face Early Industry Dissent (SCRIP-$)
  • NIB generates 3645 blood transfusion related ADRs under HvPI (PharmaBiz)


  • Summary Safety Review - ULORIC (febuxostat) - Assessing the Potential Risk of Heart Failure (Health Canada)


  • Medicines Safety Update, Volume 7, Number 2, April 2016 (TGA)


  • Zika: A Research And Development Cat And Mouse Game (SCRIP-$)
  • Zika mosquito's habits force new strategy by U.S. cities, states (Reuters)
  • CDC Warns Local Health Agencies to Prepare for Zika Virus (WSJ-$)

Other International

  • WHO Calls for Early Diagnostic Tests for Lassa Fever (ICT)
  • Boy tests positive for Ebola in latest Liberia flare-up (Reuters) (WHO)
  • As Ebola flares up, Guinea plans to vaccinate contacts of survivors (Reuters)

General Health & Other Interesting Articles

  • Andrew Wakefield's Film Fizzles (Forbes)
  • Vaccine Adjuvant Protects Against Post-Burn Infection (ICT)
  • Study results show bypass surgery extends lives of patients with heart failure (NIH) (Washington Post)

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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